FDA Tracker: Zealand’s GLP-2 Drug Rejected While Xcovery Wins First Approval, Targets NSCLC

The FDA has granted some significant approvals so far in 2024, including Eli Lilly’s Kisunla for Alzheimer’s disease and Madrigal Pharmaceuticals’ Rezdiffra for metabolic dysfunction-associated steatohepatitis. But it’s not always good news that companies have to deliver to their stakeholders; this year has also had its fair share of Complete Response Letters.

BioSpace is committed to keeping you up-to-date on all the FDA’s actions in this FDA Decision Tracker.

December 20

Product: Zealand’s glepaglutide

Indication: Short bowel syndrome

The FDA on Thursday shot down Zealand Pharma’s aspirations to use its investigational GLP-2 analog glepaglutide for the treatment of short bowel syndrome.

As per the regulator’s Complete Response Letter, the FDA asked for additional evidence to confirm the efficacy and safety of glepaglutide at its proposed dose and recommended that Zealand run another clinical trial to generate these data.

In a statement, Zealand CMO David Kendali said that the company is “certainly disappointed” by the rejection but remains “confident” in glepaglutide and the evidence supporting its use in short bowel syndrome. Zealand will continue working with the FDA to “align on the path toward regulatory approval,” he said. The biotech is also eyeing a regulatory submission to the European Medicines Agency next year.

Short bowel syndrome is characterized by having a truncated or damaged small intestine, leaving patients unable to adequately absorb nutrients from their food and typically dependent on parenteral support. Zealand is proposing to use glepaglutide to treat these patients, supporting its regulatory bid with data from one randomized, placebo-controlled late-stage trial. Twice-weekly glepaglutide significantly beat placebo at reducing patients’ parenteral support needs, though the once-weekly dosing regimen failed to achieve statistical significance.

December 18

Product: Xcovery’s Ensacove

Indication: ALK-positive locally advanced or metastatic non-small cell lung cancer

Florida-based Xcovery Holdings on Thursday scored its first FDA approval with the regulatory greenlight for Ensacove (ensartinib) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who are also positive for the anaplastic lymphoma kinase (ALK) fusion gene. Ensacove is approved for use in the first-line setting.

The ALK is a gene largely involved in development and, under healthy circumstances, is turned off before birth. In some cases, however, the gene is reactivated and fuses with neighboring genes—a mutation that can give rise to different types of cancers such as lymphoma and NSCLC.

Ensacove, an orally available tyrosine kinase inhibitor, addresses ALK-positive NSCLC by disrupting ALK-mediated signaling, in turn blocking the hyperactive growth and proliferation typical of cancer cells.

Xcovery studied Ensacove in the Phase III eXalt3 study, data from which were published in JAMA Oncology in September 2021. In the trial, Ensacove significantly improved progression-free survival than Pfizer’s Xalkori (crizotinib).

December 18

Product: Celltrion’s Steqeyma

Indication: Plaque psoriasis and psoriatic arthritis in adults and children, Crohn’s disease and ulcerative colitis in adults.

Johnson & Johnson will face even stiffer competition for its blockbuster immunotherapy Stelara (ustekinumab) after the FDA on Wednesday signed off on Celltrion’s biosimilar Steqeyma.

Like its branded reference product, Steqeyma can be used to treat adult and pediatric patients with plaque psoriasis and psoriatic arthritis, and adults with Crohn’s disease and ulcerative colitis. Steqeyma hit U.S. shelves in February 2025 and will be available as a subcutaneous injection and an intravenous infusion.

According to Celltrion, Steqeyma was approved based in part on the findings of a Phase III study in plaque psoriasis, which demonstrated that the biosimilar had no clinically meaningful differences versus Stelara in terms of efficacy or safety.

Steqeyma is the latest addition to a growing list of Stelara biosimilars. In April, the FDA also greenlit Teva and Alvotech’s Selarsdi, which can be used for plaque psoriasis and psoriatic arthritis but not for Crohn’s disease and ulcerative colitis. Selarsdi will launch in early 2025. A few months later, in July, the regulator cleared Samsung Bioepis’ Pyzchiva, which has also been granted provisional determination for interchangeability. Pyzchiva will be commercialized in the U.S. by Sandoz starting February 22, 2025.

December 18

Product: AstraZeneca’s Andexxa

Indication: Reversal of anticoagulation

The FDA has declined to give traditional approval to AstraZeneca’s recombinant factor Xa therapy Andexxa, according to reporting from Pink Sheet last week. The pharma has confirmed the rejection to various media outlets, while noting that the drug will remain available to patients.

Andexxa won the FDA’s accelerated approval in 2018 to stem bleeding in patients who had undergone anticoagulation treatment with rivaroxaban or apixaban. To confirm Andexxa’s clinical benefit AstraZeneca used data from the Phase IV ANNEXA-I study, which found that the drug could induce significantly better hemostasis in patients with acute intracranial bleeding than usual care.

However, at a meeting of the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee last month, outside experts found data from ANNEXA-I wanting, particularly its focus on a specific group of patients who needed coagulation support. Panelists found it difficult to use ANNEXA-I’s findings to make regulatory recommendations that would affect a much broader set of patients who would have access to Andexxa.

In a briefing document ahead of the committee meeting, the FDA’s internal reviewers flagged an excess risk of thrombosis and related deaths in patients treated with Andexxa versus standard care.

December 17

Product: Galderma’s Nemluvio

Indication: Moderate-to-severe atopic dermatitis

The FDA on Monday handed out a pair of atopic dermatitis nods, one of which went to Galderma and its subcutaneous injection Nemluvio (nemolizumab-ilto), which is indicated for patients aged 12 years and above who have moderate-to-severe atopic dermatitis.

According to its label, Nemluvio is recommended to be given alongside topical corticosteroids and/or calcineurin inhibitors.

Nemluvio is a humanized IgG2 monoclonal antibody that targets the IL-31 receptor and disrupts its downstream signaling, in turn dampening inflammation, fibrosis and dysregulation of the epidermis. Its approval was supported by the Phase III ARCADIA clinical trial program, which included more than 1,700 patients and found that Nemluvio given every four weeks led to significant improvements in skin clearance versus placebo.

Nemluvio also significantly alleviated itch and reduced sleep disturbances as compared with placebo.

Galderma is eyeing peak sales of over $2 billion for Nemluvio, which the biotech anticipates to hit blockbuster status by the end of 2027.

December 17

Product: Organon’s Vtama

Indication: Atopic dermatitis

Months ahead of its adjusted PDUFA date of March 12, 2025, Organon on Monday reported that the FDA has approved its topical cream Vtama (taparinof) for the treatment of atopic dermatitis. Vtama can be used in adult patients and in children as young as 2 years.

Vtama is an aryl hydrocarbon receptor agonist, the exact mechanism of action for which has yet to be completely elucidated, according to the drug’s label. Vtama was first approved in May 2022 for plaque psoriasis.

Organon supported Vtama’s application with findings from the ADORING pivotal trials, which established the cream’s significant superiority versus vehicle in terms of helping patients achieve clear or almost clear skin. Additionally, a long-term extension study established that Vtama’s effects were durable and its tolerability profile remained favorable over 48 weeks of follow-up.

Of Vtama’s approval, Organon CEO Kevin Ali said in a statement that “there is now a therapy that offers the potential for powerful skin clearance with no label warnings or precautions, contraindications, and no restrictions on duration of use or percentage of body surface area affected.”

December 16

Product: Neurocrine’s Crenessity

Indication: Congenital adrenal hyperplasia

With an early approval—coming two weeks before its PDUFA date—Neurocrine’s Crenessity (crinecerfont) on Friday became the industry’s first new congenital adrenal hyperplasia (CAH) drug in seven decades.

CAH is a rare genetic disease characterized by deficiencies in important adrenal steroid hormones, including cortisol, aldosterone and adrenal androgens. Patients with CAH suffer from dehydration and vomiting, and in severe cases, the disease leads to salt wasting and even death. Prior to Crenessity’s approval, patients and their providers managed symptoms with glucocorticoids, which can help correct enzyme imbalances but can come with side effects such as osteoporosis and diabetes.

Crenessity works by selectively blocking the CRF1 receptor to help temper excess ACTH and other adrenal androgens. Its approval was supported by data from the Phase III CAHtalyst study, which demonstrated that Crenessity significantly lowered androstenedione concentrations in both adult and pediatric patients, allowing them to taper glucocorticoid doses without affecting treatment efficacy.

BMO Capital Markets analysts expect a slow launch, however, writing in a note to investors about challenges including a black box warning for “risk of acute adrenal insufficiency or adrenal crisis with inadequate concomitant glucocorticoid therapy.” The firm said it anticipates broad commercial access will not happen until the second half of 2025.

December 16

Product: Checkpoint Therapeutics’ Unloxcyt

Indication: Metastatic or locally advanced cutaneous squamous cell carcinoma

The FDA on Friday signed off on the use of Checkpoint Therapeutics’ PD-L1 inhibitor Unloxcyt for the treatment of patients with metastatic or locally advanced cutaneous squamous cell carcinoma.

According to Checkpoint, Unloxcyt is the “first and only” PD-L1 blocker indicated for this indication, with CEO James Oliviero saying in a statement that the biotech expects to “compete in a U.S. market estimated to exceed $1 billion annually.” Compared with other approved drugs on the market, Unloxcyt could be a differentiated option by targeting PD-L1 rather than PD-1 and because it can induce antibody-dependent cell-mediated cytotoxicity, Oliviero added.

Unloxcyt’s approval is backed by pivotal data, which demonstrated confirmed objective response rates of 47.4% and 54.8% in patients with metastatic and locally advanced disease, respectively.

The approval also comes after a previous rejection in December 2023 linked to problems with a third-party contract manufacturer.

December 5

Product: AstraZeneca’s Imfinzi

Indication: Limited-stage small cell lung cancer

AstraZeneca on Wednesday successfully expanded the label of its PD-L1 blocker Imfinzi (durvalumab), notching the FDA’s greenlight for its use in patients with limited-stage small cell lung cancer who have not progressed after concurrent platinum chemotherapy and radiation therapy.

The approval was supported by data from the Phase III ADRIATIC study, which according to an April 2024 readout hit its primary efficacy endpoints of overall survival and progression-free survival (PFS). While AstraZeneca at the time did not disclose specific data, the FDA in its news release on Wednesday said treatement with Imfinzi reduced the risk of death by 27% versus placebo, while also improving PFS by 24%. Both effects were statistically significant.

In August, the FDA also cleared Imfinzi for the perioperative treatment of patients with non-small cell lung cancer—despite growing skepticism about the need for PD-L1 exposure both before and after surgery.

December 5

Product: Merus’ Bizengri

Indication: Pancreatic adenocarcinoma and non-small cell lung cancer with NGR1 mutations

Merus’ bispecific antibody zenocutuzumab on Wednesday became the “first and only” therapy for advanced unresectable or metastatic pancreatic adenocarcinoma and NSCLC with NRG1 gene fusions. The FDA approved zenocutuzumab—now to be marketed as Bizengri—under its accelerated pathway. Merus will need to verify Bizengri’s clinical benefit in a confirmatory trial to keep it on themarket.

Bizengri works by binding to the extracellular domains of HER2 and HER3, both of which are typically expressed on tumor cells. This mechanism of action prevents the dimerization of these two proteins and blocks the binding of NRG1 to HER3, in turn reducing cell proliferation and promoting the immune system’s cancer-killing activity.

The FDA’s accelerated approval was backed by the eNRGy study, which touted a 40% and 33% overall response rate in patients with pancreatic adenocarcinoma and NSCLC, respectively. Duration of response in the former patient group reached up to 16.6 months, while the latter group had a median duration of 7.4 months.

December 3

Product: Biocon Biologics’ Yesintek

Indication: Crohn’s disease, ulcerative colitis, plaque psoriasis and psoriatic arthritis

The FDA on Monday signed off on Biocon Biologics’ Yesintek (ustekinumab-kfce), a biosimilar to Johnson & Johnson’s blockbuster antibody Stelara.

Like its reference product, Yesintek is indicated for plaque psoriasis and psoriatic arthritis, as well as the inflammatory bowel diseases Crohn’s disease and ulcerative colitis. As per a February 2024 settlement agreement with J&J, Biocon will be able to launch Yesintek In the U.S. no later than February 22, 2025.

With Monday’s approval, Yesintek joins a growing group of Stelara copycats poised to challenge the blockbuster drug next year. These include Teva and Alvotech’s Selarsdi, approved in April 2024, and Samsung Bioepis’ Pyzchiva, which will be commercialized by Sandoz. Adding to the pressure on J&J is a September 2024 announcement from Cigna’s Evernorth Health Services that it will offer an interchangeable biosimilar alternative to Stelara at $0 out-of-pocket.

November 28

Product: Applied Therapeutics’ govorestat

Indication: Galactosemia

Three months after its original target action date, the FDA denied approval of Applied Therapeutics’ govorestat for the rare, genetic metabolic disorder galactosemia. In its Complete Response Letter, the regulator cited “deficiencies” in the company’s clinical application.

In a Nov. 28 note to investors, William Blair analysts wrote that Applied Therapeutics “suspects” the CRL “was related to the efficacy package as opposed to [chemistry, manufacturing and controls] or safety.”

Results from the Phase III ACTION-Galactosemia Kids study, unveiled in April 2023, showed that govorestat fell short of its primary efficacy endpoint, a composite of four clinical measures designed to assess various domains of galactosemia symptoms. However, in the Phase I/II ACTION-Galactosemia study, which focused on adults, treatment with govorestat resulted in a drop in galactitol concentrations, according to Applied Therapeutics.

The FDA had set an initial target date of Aug. 28 for govorestat, but in March pushed this back, saying it needed more time to review supplemental submissions.

While the company was “disappointed” by the rejection, Applied CEO Soshana Shendelman said in a statement that it plans to “work with the FDA to address concerns in the CRL and determine an expeditious path” forward for govorestat.

November 27

Product: Orphan Pharmaceuticals’ Rapiblyk

Indication: Atrial fibrillation and atrial flutter in critical care

Vienna-based AOP Orphan Pharmaceuticals on Wednesday won the FDA’s approval for Rapiblyk (landiolol) for the treatment of atrial fibrillation and atrial flutter in the critical care setting.

Designed to be administered intravenously, Rapiblyk is an ultra-short-acting blocker of the adrenergic receptor, the activity of which is known to affect heart rate. According to AOP, Rapiblyk has a “fast onset of action,” resulting in the rapid decrease in heart rate without significantly suppressing patients’ blood pressure.

The FDA’s decision is backed by results from five randomized placebo-controlled trials, which together demonstrated a 40% to 90% reduction in heart rate within 10 minutes of dosing, versus 0% to 11% in placebo counterparts. Adverse events were more common in Rapiblyk-treated patients than in placebo.

Rapiblyk is intended for use in the emergency and critical care departments, as well as in the operating and intensive care settings. It is not intended for the treatment of chronic cardiac arrhythmia.

November 25

Product: Shorla Oncology’s Imkeldi

Indication: Several forms of leukemia and other cancers

The FDA on Monday signed off on Shorla Oncology’s Imkeldi, an oral solution indicated for the treatment of certain types of chronic myeloid leukemia and acute lymphoblastic leukemia, as well as other cancers such as myelodysplastic/myeloproliferative diseases, aggressive systemic mastocytosis and dermatofibrosarcoma protuberans.

Imatinib, the active ingredient in Imkeldi, is a tyrosine kinase inhibitor that works by blocking the BCR-ABL protein, in turn preventing the uncontrolled growth of cancer cells and triggering their death. It was initially approved in 2001 and is sold by Novartis as tablets under the brand name Gleevec.

Compared with other forms of imatinib, Imkeldi’s liquid formulation provides better dosing accuracy, which CEO Sharon Cunningham said can “ensure more precise and consistent dosing.” For patients who have difficulty swallowing pills or need specific doses according to the affected body surface area, Imkeldi can offer a “convenient alternative to compounding,” Cunningham added.

According to Shorla, Imkeldi is the first FDA-approved orally available liquid formulation of imatinib for certain leukemias.

November 22

Product: BridgeBio’s Attruby

Indication: Transthyretin amyloid cardiomyopathy

Thanksgiving came early for BridgeBio—and transthyretin amyloid cardiomyopathy (ATTR-CM) patients—after the FDA approved the company’s Attruby a week before its scheduled PDUFA date.

Attruby’s approval was supported by positive results from the Phase III ATTRibute-CM, where the transthyretin stabilizer significantly reduced death and cardiovascular-related hospitalization, and improved quality of life.

Attruby is the “first and only approved product with a label specifying near-complete stabilization of TTR,” according to a press release issued Friday by BridgeBio.

Analysts agree that the drug’s broad label—which states a reduction in the likelihood of both hospitalization and death—will help it in the competition against Pfizer’s already-established tafamidis franchise and Alnylam’s Amvuttra, the supplemental New Drug Appilcation for which was accepted by the FDA on Monday. The all-cause mortality benefit had been in question after Attruby missed this secondary endpoint in ATTRibute-CM.

November 22

Product: Generic exenatide

Indication: Type 2 diabetes

The highly lucrative and competitive GLP-1 space on Friday welcomed one of its first generic entrants with the FDA’s approval of Amneal Pharmaceuticals’ exenatide, a copycat version of AstraZeneca’s Byetta.

Like its branded reference product, Amneal’s generic version is indicated for the improvement of glucose control in type 2 diabetes patients, combined with diet and exercise. Generic exenatide will be available in 1.2-mL and 2.4-mL prefilled pens. Similarly, the copycat drug shares the adverse effect profile of Byetta, which includes nausea, vomiting, dizziness, diarrhea and low blood sugar, among other common side effects.

Amneal’s exenatide is “one of the first generic GLP-1 injectables to be approved in the U.S.,” Chief Commercial Officer Andy Boyer said in a statement. According to a Wednesday report by Coherent Marketing Insights, the GLP-1 market is expected to hit around $55.7 billion by 2031. Currently, the space is dominated by Eli Lilly and Novo Nordisk, which market tirzepatide and semaglutide, respectively, and which have effectively cornered the diabetes and obesity markets for GLP-1 drugs.

Outside the U.S., generics developers have already started to threaten these companies’ profits, particularly in China and India.

November 21

Product: Jazz and Zymeworks’ Ziihera

Indication: HER2-positive biliary tract cancer

With the FDA’s approval of Ziihera on Thursday, Jazz Pharmaceuticals and Zymeworks will go toe-to-toe with AstraZeneca and Daiichi Sankyo, whose antibody-drug conjugate Enhertu (fam-trastuzumab deruxtecan-nxki) was given a broad tumor-agnostic approval in April 2024.

Ziihera is a bispecific antibody that targets two non-overlapping sites on the HER2 receptor. This unique binding profile gives Ziihera several mechanisms of action, including the dual blockage of HER2 signaling and removal of HER2 proteins on the surface of cancer cells. The bispecific also boosts the anti-cancer activity of the immune system.

Thursday’s regulatory victory is backed by data from the Phase IIb HERIZON-BTC-01 study, which reported a 41.3% confirmed objective response rate in patients treated with Ziihera, with treatment response lasting for a median of 12.9 months. As for safety, the mid-stage trial found Ziihera to be well-tolerated, with only two patients discontinuing treatment due to toxicities.

Ziihera is approved under the FDA’s accelerated pathway. To keep it on the market, Jazz and Zymeworks are running the Phase III HERIZON-BTC-302 confirmatory trial, which is assessing Ziihera in the first-line setting, in combination with chemotherapy with or without a PD-(L)1 blocker.

November 20

Product: Bimzelx

Indication: Moderate-to-severe hidradenitis suppurativa

UCB on Wednesday won the FDA’s approval to use its subcutaneous injection Bimzelx (bimekizumab-bkzx) to treat adult patients with moderate-to-severe hidradenitis suppurativa.

Bimzelx is a humanized IgG1 monoclonal antibody designed to target the IL-17A and IL-17F cytokines, which are known to play key roles in immune and inflammatory cascades. In October 2023, the FDA cleared the use of Bimzelx for adult patients with moderate-to-severe plaque psoriasis and in September 2024 expanded its label to include psoriatic arthritis, ankylosing sspondylitis and non-radiographic axial spondyloarthritis.

Wednesday’s approval is backed by data from two Phase III studies—BE HEARD I and BE HEARD II. Taken together, results from these trials showed that more patients treated with Bimzelx saw at least a 50% improvement in the signs and symptoms of hidradenitis suppurativa at 16 weeks, as compared with placebo. This treatment response was maintained through 48 weeks of follow-up. Bimzelx also induced clinically meaningful improvements in several key secondary outcomes.

Bimzelx is the first and so far only approved therapy that selectively blocks IL-17A and IL-17A, according to UCB.

November 20

Product: Izervay

Indication: Geographic atrophy secondary to age-related macular degeneration

The FDA on Tuesday turned down Astellas Pharma’s bid to include two-year data on the label of its eye injection Izervay (avacincaptad pegol intravitreal solution).

Izervay is a PEGylated oligonucleotide that works by targeting and disabling the C5 complement protein, thus addressing an underlying cause of age-related macular degeneration (AMD). It was approved in 2023 for geographic atrophy (GA) secondary to AMD, but its current label only recommends treatment for up to 12 months.

In April 2024, Astellas filed a supplemental New Drug Application with the FDA, seeking to add two-year data to Izervay’s label. The pharma used findings from the Phase III GATHER2 trial, which showed that Izervay continued to reduce the growth of GA lesions through 24 months versus a sham control.

In its Complete Response Letter on Tuesday, the FDA focused on a “statistical matter related to labelling language,” as proposed by Astellas. The regulator did not find any issues with the safety or benefit/risk profile of Izervay through two years. The pharma is currently “seeking further clarification” for the rejection, with an eye toward quickly addressing the FDA’s comments, according to its news release.

November 15

Product: Revuforj

Indication: Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation

Syndax Pharmaceuticals’ Revuforj (revumenib) on Friday became the market’s first menin inhibitor for relapsed or refractory acute leukemia when the FDA cleared it for use in adult or pediatric patients carrying a translocation mutation in the lysine methyltransferase 2A (KMT2A) gene.

In acute leukemia, KMT2A gene rearrangements are linked to more aggressive disease characterized high rates of relapse and very poor prognoses. Among patients with KMT2A rearrangement mutations, approximately half will relapse after conventional therapies, according to Syndax. Nearly all of KMT2A rearrangements are classified as translocation mutations, which produce a fusion protein that binds to the menin protein, triggering the development of leukemia.

Revuforj’s approval was supported by data from 104 patients enrolled in the Phase I/II AUGMENT-101 study, in which 21% of treated patients reached complete remission or complete remission with partial hematological recovery. This type of treatment response had a median duration of 6.4 months.

Syndax expects to make 110-mg and 160-mg tablets of Revuforj available in the U.S. this month. Meanwhile, the 25-mg dose, which can be used for young patients or those weighing less than 40 kg, will be available in late first quarter or early second quarter next year.

November 14

Product: Danziten tablets

Indication: Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia

Azurity Pharmaceuticals on Thursday won the first FDA approval for oral nilotinib without mealtime restrictions.

Nilotinib is an orally available inhibitor of the BCR-ABL kinase and works by preventing the growth and proliferation of cancer cells, in turn inducing their death. Nilotinib is approved under the brand name Tasigna, which is owned and marketed by Novartis, and is indicated for Philadelphia chromosome-positive chronic myeloid leukemia.

According to Azurity, however, Tasigna’s bioavailability spikes when taken with food and can lead to cardiac side effects. Patients on Tasigna often need to adhere to strict fasting before taking the drug to avoid these toxicities. As a workaround, Azurity re-engineered nilotinib to produce Danziten, which has improved bioavailability than Tasigna, allowing for reduced dosing and eliminating the requirement for mealtime restrictions.

Azurity expects to make Danitzen available “in the coming weeks,” according to its announcement.

November 12

Product: Intercept Pharmaceuticals’ Ocaliva

Indication: Primary biliary cholangitis

After initially delaying its decision last month, the FDA on Tuesday declined to give Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) full approval for primary biliary cholangitis (PBC).

Ocaliva, a farsenoid X receptor agonist, won the FDA’s accelerated approval for PBC in May 2016. In September 2024, however, the regulator’s Gastrointestinal Drugs Advisory Committee nearly unanimously voted against the oral drug, noting that Intercept had not provided enough data to support Ocaliva’s clinical benefit. In a briefing document released prior to the panel discussion, the FDA’s internal reviewers said that Ocaliva had “failed to demonstrate efficacy” in one of its confirmatory trials.

In its Complete Response Letter on Tuesday, the FDA indicated that it was continuing to review safety data for Ocaliva from one of its confirmatory studies. In the meantime, the drug will remain available to PBC patients under its accelerated approval status, according to Intercept.

November 8

Product: Autolus Therapeutics’ Aucatzyl

Indication: B cell acute lymphoblastic leukemia

Aucatzyl joins the still-limited roster of FDA-approved CAR T therapies after the regulator on Friday signed off on its use for the treatment of relapsed or refractory B cell acute lymphoblastic leukemia (ALL) in adults.

Autolus backed Aucatzyl with data from the Phase II FELIX study, which showed a 63% overall complete remission rate, including 51% of patients who achieved complete remission. Aucatzyl’s edge appears to be its safety, with a low incidence of cytokine release syndrome and grade 3 toxicities, as well as no grade 4 or 5 adverse events.

“We continue to believe the more tolerable safety profile of Aucatzyl will make it the preferred treatment option for patients with adult ALL and support majority market share over time,” William Blair analyst Matt Phipps said in a Friday note. Aucatzyl is the first CAR T therapy to be approved without a Risk Evaluation Mitigation Strategy program requirement.

November 4

Product: Journey Medical’s Emrosi

Indication: Inflammatory lesions of rosacea

Emrosi’s journey to FDA approval is complete after the FDA approved the oral medicine to treat inflammatory lesions of rosacea on Monday.

“Rosacea is a difficult to treat skin condition and based on the favorable results from our Phase 3 clinical trials, Emrosi has potential to become the best-in-class oral medication to treat the condition,” Claude Maraoui, CEO of Arizona-based Journey Medical said in a statement.

Emrosi’s approval is supported by two Phase III trials, in which the treatment met all co-primary and secondary endpoints. Emrosi demonstrated “statistically significant superiority” over the current standard-of-care treatment (Oracea 40-mg capsules) and placebo for Investigator’s Global Assessment treatment success and reduction in total inflammatory lesion count in both trials, Journey Medical reported.

October 29

Product: Novartis’ Scemblix

Indication: Chronic myeloid leukemia

Novartis’ Scemblix is now a first-line treatment option for certain patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (CML) in the chronic phase after the FDA granted a label expansion Tuesday. The new approval broadens the eligible patient population for Scemblix by approximately four times, according to Novartis.

An orally available tyrosine kinase inhibitor that works by blocking the BCR-ABL1 fusion protein, Scemblix received accelerated approval in November 2021 for patients with Philadelphia chromosome-positive, chronic phase CML and full approval for patients in this population whose cancer carries the T315I mutation.

The label expansion was supported by data from the Phase III ASC4FIRST study, in which 68% of patients treated with Scemblix achieved a major molecular response compared with 49% of those treated with standard-of-care tyrosine kinase inhibitors, including imatinib and nilotinib.

October 29

Product: Shorla Oncology’s Jylamvo

Indications: Pediatric acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis

Shorla Oncology received expanded approval of Jylamvo Tuesday for pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA). Jylamvo is the only oral liquid methotrexate on the market approved for both adult and pediatric indications, according to the U.S.-Ireland specialty pharmaceutical company.

Jylamvo first won approval in November 2022 to treat adults with acute lymphoblastic leukemia, mycosis fungoides, relapsed or refractory non-Hodgkin lymphoma, rheumatoid arthritis and severe psoriasis.

“This approval follows Jylamvo’s successful use in adult patients and represents a critical step forward in addressing the unmet needs of pediatric care in oncology and autoimmune diseases,” Shorla CEO Sharon Cunningham said in a statement.

October 29

Product: Protega Pharmaceuticals’ Roxybond

Indication: Severe pain

Protega Pharmaceuticals secured approval Tuesday for Roxybond 10-mg tablets for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. Previously approved in 5-mg, 15-mg and 30-mg tablets, Roxybond is the first and only FDA-approved abuse-deterrent immediate-release opioid, according to the company.

Roxybond leverages Protega’s SentryBond abuse-deterrent technology, which combines inactive excipients with active pharmaceutical ingredients to make the tablet more difficult to manipulate for misuse and abuse, even with physical manipulation or chemical extraction.

This latest approval of Roxybond “fulfills an unmet need for an IR (immediate-release) opioid with abuse-deterrent technology that may reduce misuse and abuse while providing pain relief to medically appropriate patients when used as indicated,” Paul Howe, chief commercial officer of Protega, said in a statement.

The 10-mg option can “enhance flexibility and precision in opioid therapy,” according to Protega, “aiming to support both physicians and patients in achieving more effective and safer pain management outcomes.”

October 25

Product: Iterum Therapeutics’ Orlynvah

Indication: Urinary tract infections

Iterum Therapeutics has its first approved product and women with uncomplicated urinary tract infections have another treatment option after the FDA greenlit the company’s Orlynvah on Friday.

The first oral penem to receive approval in the U.S., Orlynvah is cleared for three designated microorganisms—Escherichia coli, Klebsiella pneumoniae and Proteus mirabilis—and is indicated for women with limited or no available alternative oral antibiotic options.

Orlynvah’s approval was supported by two Phase III trials. In the pivotal late stage REASSURE study, Iterum’s drug showed statistical non-inferiority to Augmentin (amoxicillin/clavulanate) in terms of overall response, and in the pivotal Phase III SURE 1 study, Orlynvah outperformed ciprofloxacin in patients with quinolone-resistant infections. Safety outcomes were comparable between treatment groups in both studies.

October 22

Product: Pfizer’s Abrysvo

Indication: Respiratory syncytial virus

Younger adults at higher risk of respiratory syncytial virus (RSV)–related lower respiratory tract disease now have a prophylactic option after the FDA expanded the label for Pfizer’s Abrysvo on Tuesday. Abrysvo is the first RSV shot that can be used in adults under 50 years of age, according to Pfizer.

The label expansion was backed by Abrysvo’s “inferred efficacy” in the Phase III MONeT trial. The trial met its primary efficacy endpoint, eliciting a non-inferior immune response in younger adults as in participants aged 60 years and above, according to topline data revealed by Pfizer in April.

Abrysvo is also the only vaccine option for pregnant women from 32 through 36 weeks of gestation, intended to elicit protection in infants from birth through six months.

October 22

Product: Camurus’ CAM2029

Indication: Acromegaly

Manufacturing problems tripped up Camurus’ application for CAM2029, an investigational subcutaneous formulation of octreotide the Swedish company is developing acromegaly. A rare hormonal disorder, acromegaly is often caused by a tumor in the pituitary gland.

In its Complete Response Letter, the FDA pointed to “facility-related deficiencies” discovered last month during an inspection of a third-party manufacturer as the reason for the rejection. The regulator requires “satisfactory” responses from this third-party manufacturer before the CRL can be resolved, according to Camurus.

Camurus CEO Fredrik Tiberg said his company will work with its external manufacturer and with the FDA “to bring CAM2029 to patients living with acromegaly as soon as possible,” and noted that Camurus has already held labelling discussions with the FDA.

October 18

Product: Astellas’ Vyloy

Indication: Gastric and gastroesophageal junction cancer

There’s a new treatment option for people with certain gastric cancers after the FDA greenlit Astellas’ Vyloy on Friday. With the approval, Vyloy becomes the first claudin 18.2–targeted therapy in the U.S. for gastric or gastroesophageal junction cancer, according to Astellas.

Vyloy is specifically approved for the first line treatment of claudin 18.2–positive patients with HER2-negative tumors, in combination with fluoropyrimidine- and platinum-containing chemotherapy.

The approval is backed by data from the Phase III SPOTLIGHT and GLOW studies, both of which met the primary endpoint of progression-free survival and key secondary endpoint of overall survival. In both studies, Vyloy was well-tolerated, with the most common toxicities including nausea, vomiting and weakened appetite.

October 17

Product: AbbVie’s Vyalev

Indication: Parkinson’s disease

People with advanced Parkinson’s disease have a new treatment option Thursday as the FDA greenlit AbbVie’s Vyalev as “the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations” in this patient population.

Adult patients treated with Vyalev experienced superior improvement in “on” time—periods during which patients experience optimal motor symptom control—without troublesome dyskinesia, compared to oral immediate-release carbidopa/levodopa, AbbVie reported.

“Due to the progressive nature of the disease, oral medications are eventually no longer as effective at motor symptom control and surgical treatment may be required,” Robert A. Hauser, professor of neurology and director of the Parkinson’s and Movement Disorder Center at the University of South Florida, said in a statement. “This new, non-surgical regimen provides continuous delivery of levodopa morning, day and night.”

October 16

Product: Avadel Pharmaceuticals’ Lumryz

Indication: Cataplexy or excessive daytime sleepiness associated with narcolepsy in children

Avadel Pharmaceuticals’ Lumryz is now approved to treat pediatric patients 7 years of age and older with excessive daytime sleepiness (EDS) or cataplexy (sudden muscle weakness), both symptoms of narcolepsy. Lumryz was first approved for adults in 2023.

With the FDA nod, Lumryz becomes the only FDA-approved once-at-bedtime oxybate treatment for cataplexy and EDS in this patient population.

“This approval represents an important milestone for the narcolepsy community, specifically for younger narcolepsy patients and their caregivers who face significant challenges associated with waking up in the middle of the night to complete treatment regimens,” Greg Divis, CEO of Avadel, said in a statement.

Lumryz was granted Orphan Drug Exclusivity in this population through Oct. 16, 2031.

October 11

Product: Pfizer’s Hympavzi

Indication: Hemophilia A and hemophilia B

Friday, the FDA approved Pfizer’s Hympavzi as the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S. Hympavzi, which was simultaneously authorized for the treatment of hemophilia A, is also the first treatment available through a pre-filled pen or syringe for either condition, according to Pfizer. Hympavzi is intended for routine prophylaxis to lower the frequency of or completely prevent bleeding episodes in patients with hemophilia.

Approval of Hympavzi was supported by data from the Phase III BASIS trial, which enrolled nearly 190 patients with severe hemophilia A or moderately severe to severe hemophilia B. In this trial, treatment with Pfizer’s drug led to a 92% reduction in bleeding in patients lacking inhibitors to factor VIII or factor IX; annual bleeding rate dropped by 35%.

October 9

Product: Zealand Pharma’s dasiglucagon

Indication: Congenital hyperinsulinism

Timing is everything, if you’re Zealand Pharma. The Denmark-based company announced Wednesday it had received a Complete Response Letter for dasiglucagon, which it was proposing for the ultra-rare genetic disease congenital hyperinsulinism, “due to the timing of a third-party manufacturing facility reinspection.”

In an initial visit, the FDA had flagged “deficiencies” in a third-party contract manufacturer, which the company claims had been resolved by the time of a reinspection in August/September 2024. However, Zealand’s service provider has yet to receive its inspection classification. The agency did not note any concerns about the clinical data package or safety of dasiglucagon, according to Zealand.

Noting the “no or very limited treatment options” for congenital hyperinsulinism, Zealand Chief Medical Officer David Kendall said the company is “committed to working with the FDA and our third-party manufacturing partner to bring dasiglucagon to patients living with this devastating disease in the months ahead.”

September 30

Product: Fresenius Kabi and Formycon’s Otulfi

Indication: Crohn’s disease, ulcerative colitis, plaque psoriasis and active psoriatic arthritis

There’s a new Stelara biosimilar on the block after the FDA greenlit Otulfi, from partners Fresenius Kabi and Formycon, for the treatment of Crohn’s disease, ulcerative colitis, moderate to severe plaque psoriasis and active psoriatic arthritis—all conditions for which the reference product is also approved.

The FDA’s approval, which is for both subcutaneous and intravenous formulations of Otulfi, was backed by a “thorough evaluation of a comprehensive data package,” which included analytical, preclinical, clinical and manufacturing data, according to the companies.

Fresenius Kabi is allowed to begin marketing Otulfi—its fourth biosimilar approved in the U.S.—no later than Feb. 22, 2025.

September 27

Product: Sanofi and Regeneron’s Dupixent

Indication: Chronic obstructive pulmonary disease

Sanofi and Regeneron won a seventh indication for their blockbuster drug Dupixent Friday, as it became the first biologic medicine in the U.S. for chronic obstructive pulmonary disease (COPD).

The pharma partners backed their application for Dupixent in COPD with data from a pair of Phase III trials, in which treatment with the injection elicited a 30% and 34% reduction in the annualized rate of moderate or severe COPD exacerbations compared to placebo. Dupixent also led to statistical improvements in post-bronchodilator forced expiratory volume levels from baseline at week 12, satisfying the trials’ secondary endpoint.

Safety was consistent with Dupixent’s profile in its other approved indications, which include asthma and eczema.

Read more here.

September 26

Product: Bristol Myers Squibb’s Cobenfy

Indication: Schizophrenia

BMS made recent history on Thursday with the approval of Cobenfy—formerly KarXT—as the first novel therapeutic for schizophrenia in 35 years. While treatment for schizophrenia up until now has focused on blocking dopamine signaling across the brain, Cobenfy targets the M1 and M4 muscarinic receptors.

Cobenfy’s approval was supported by data from three registrational trials in which the drug showed benefit in treating schizophrenia on the Positive and Negative Syndrome Scale.

The drug, which was originally developed at Karuna Therapeutics, began its development journey as an investigational treatment at Eli Lilly for Alzheimer’s disease, where its effects on the psychotic symptoms of the disease where discovered. BMS picked up the drug in its December 2023 acquisition of Karuna.

Read more here.

September 26

Product: AstraZeneca’s Tagrisso

Indication: Non-small cell lung cancer

AstraZeneca expanded the reach of its non-small cell lung cancer (NSCLC) drug Tagrisso Thursday as the FDA greenlit the treatment for adults with unresectable, Stage III epidermal growth factor receptor-mutated (EGFRm) disease that has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy. Tagrisso is specifically indicated for people with exon 19 deletions or exon 21 mutations.

The FDA’s approval was supported by results of the Phase III LAURA trial, in which treatment with Tagrisso reduced the risk of disease progression or death by 84% compared to placebo. Tagrisso also extended progression-free survival by 33.5 months over placebo, according to AstraZeneca.

Suresh Ramalingam, executive director of Winship Cancer Institute of Emory University and the trial’s principal investigator, called the approval a “major breakthrough” for this patient group.

Tagrisso is also approved to treat NSCLC with EGFR mutations in the first line metastatic setting as a monotherapy and in combination with chemotherapy, and as an adjuvant treatment for early-stage disease.

September 24

Product: IntraBio’s Aqneursa

Indication: Niemann-Pick disease type C

A week ago, there were no FDA-approved treatments for Niemann-Pick disease type C (NPC). Now, there are two. Days after the regulator approved Zevra Therapeutics’ Miplyffa for the ultrarare neurodegenerative disease, it gave the green light to IntraBio’s Aqneursa. The drug is specifically indicated to ease neurological symptoms in both adults and children weighing at least 15 kg.

Aqneursa’s approval was backed by data from a randomized, double-blind and placebo-controlled crossover study. Over a 12-week period, participants showed better functional performance as measured by a score that assesses their gait, sitting, stance and speech. Aqneursa was well-tolerated, with common side effects being vomiting, abdominal pain, difficulty swallowing and upper respiratory tract infections.

The second NPC approval in a week “highlights” the FDA’s “commitment” to supporting the development of innovative therapies for rare diseases, Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a statement.

September 23

Product: UCB’s Bimzelx

Indication: Psoriatic arthritis, non-radiographic axial spondyloarthritis and ankylosing spondylitis

UCB’s Bimzelx was approved Monday for not one, but three chronic immune-mediated inflammatory diseases: psoriatic arthritis (PsA), non-radiographic axial spondyloarthritis (nr-axSpA) and ankylosing spondylitis (AS). For all three indications, Bimzelx is the first approved medicine designed to selectively inhibit two key inflammation-driving cytokines, interleukin 17A (IL-17A) and interleukin 17F (IL-17F), according to UCB.

The approval of Bimzelx in active PsA is backed by two Phase III studies, in which the drug showed statistically significant improvements vs. placebo after 16 weeks in both joint and skin symptoms, results that were sustained to week 52. Meanwhile, across two Phase III studies in nr-axSpA and AS, Bimzelx demonstrated statistically significant improvements vs. placebo in signs and symptoms after 16 weeks—these results were also sustained up to one year.

Bimzelx was previously approved by the FDA in October 2023 for adult patients with plaque psoriasis who are candidates for systemic therapy or phototherapy.

September 20

Product: Zevra Therapeutics’ Miplyffa

Indication: Niemann-Pick disease type C

Patients with Niemann-Pick disease type C (NPC) have their first treatment after the FDA approved Zevra Therapeutics’ Miplyffa for the ultra-rare, progressive neurodegenerative disease. Specifically, Miplyffa is authorized, in combination with miglustat, to treat the neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.

In a statement, Zevra President and CEO Neil F. McFarlane called the approval of Miplyffa a “monumental milestone” for NPC patients and their family members in the U.S.

Miplyffa’s approval was backed by data from a 12-month multicenter, randomized, double-blind, placebo-controlled trial that investigated the treatment in NPC patients 2 to 19 years of age. Results from this trial showed that Miplyffa, in combination with miglustat, halted disease progression through 12 months of treatment, as evidenced by a decrease of 0.2 points from baseline on the rescored 4-domain NPC Clinical Severity Scale. This was compared to 1.9 points of progression for patients treated with miglustat alone.

September 20

Product: AstraZeneca’s FluMist

Indication: Influenza

Just in time for the start of flu season, the FDA greenlit the first self-administered influenza vaccine. FluMist, a needle-free nasal spray, is authorized to be self-administered by adults up to 49 years of age, and by a parent or caregiver to children two to 17 years of age.

“Each year, influenza poses a significant burden for people, society and health systems; additional tools to increase access to vaccinations, while also reducing disparities, are critical,” Ravi Jhaveri, division head of infectious disease and Virginia H. Rogers professor in infectious diseases, professor of pediatrics (infectious diseases) at Northwestern University School of Medicine, said in a statement.

AstraZeneca supported its application for self-administered FluMist with results from a usability study showing that the intended populations could self-administer or administer the vaccine.

September 19

Product: Johnson & Johnson’s Rybrevant

Indication: Non-small cell lung cancer

Johnson & Johnson racked up its third non-small cell lung cancer (NSCLC) approval for Rybrevant this year as the FDA greenlit the bispecific antibody plus chemotherapy for the second-line treatment of locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 19 deletions or L858R substitution mutations. Rybrevant is specifically authorized for patients whose disease has progressed on or after treatment with an EGFR tyrosine kinase inhibitor.

The approval is backed by results from the Phase III MARIPOSA-2 trial, where treatment with Rybrevant reduced the risk of disease progression or death by 52% vs. chemotherapy alone. Median progression-free survival for patients treated with the combo regimen was 6.3 months, compared to 4.2 months for those who received chemo alone. The overall response rate for Rybrevant plus chemo was 53% compared to 29% with chemo alone.

In August, Rybrevant was approved in combination with lazertinib for the first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitution mutations. This followed a March approval for Rybrevant in combination with chemo for the first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations.

September 18

Product: AstraZeneca’s Fasenra

Indication: Eosinophilic granulomatosis with polyangiitis

AstraZeneca won approval Wednesday for Fasenra to treat eosinophilic granulomatosis with polyangiitis (EGPA), a rare, immune-mediated disease that can result in damage to multiple organs. Fasenra was first approved in 2017 to treat severe eosinophilic asthma.

The approval “demonstrates the potential of Fasenra to help patients suffering from eosinophilic disease beyond severe asthma,” Ruud Dobber, executive vice president of AstraZeneca’s biopharma business unit, said in a statement.

The FDA’s greenlight was supported by data from the Phase III MANDARA trial, which set Fasenra against GSK’s Nucala, previously the only approved treatment for EGPA. Fasenra had a slight edge on GSK’s drug, eliciting a 59% remission rate compared to 56% in the Nucala arm. While this difference was not enough to meet the superiority bar, it did establish the non-inferiority of Fasenra.

September 18

Product: Merck’s Keytruda

Indication: Pleural mesothelioma

Merck added another indication to its extensive list of Keytruda approvals Wednesday: pleural mesothelioma. Specifically, Keytruda is now authorized to treat adults with unresectable advanced or metastatic malignant pleural mesothelioma, a type of cancer that begins in the linings of the chest and lungs.

Gregory Lubiniecki, vice president of oncology clinical research at Merck Research Laboratories, noted in a statement that prognoses are generally poor for this disease and said the company is “pleased to offer a new first-line treatment option.”

The approval was backed by results from the Phase II/III KEYNOTE-483 trial, where Keytruda elicited a significant survival benefit for patients, cutting the risk of death by 21%.

September 17

Product: Novartis’ Kisqali

Indication: Breast cancer

Novartis has “approximately doubled” the eligible patient population for its Kisqali in breast cancer, as the FDA greenlit a label expansion of the drug, in combination with an aromatase inhibitor, for earlier stages of the disease.

“With this approval, we are redefining treatment options” for more breast cancer patients who are at “persistent risk” of recurrence, Victor Bultó, Novartis’ U.S. president, said in a statement.

Novartis supported the expansion application with topline data from the Phase III NATALEE trial, in which treatment with Kisqali led to a significant improvement in disease-free survival versus standard adjuvant endocrine therapy alone, according to the company. In an updated analysis released this week, Novartis reported that Kisqali showed “deepening benefit,” reducing the risk of recurrence by 28.5% in a broad population of patients with early breast cancer.

September 13

Product: Roche’s Ocrevus Zunovo

Indication: Multiple sclerosis

People with relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) have a new treatment option after the FDA greenlit Roche’s Ocrevus Zunovo Friday. Ocrevus Zunovo is the first twice-a-year 10-minute subcutaneous injection approved to treat RMS and PPMS, according to Roche.

“This approval may offer greater flexibility for healthcare providers and people living with multiple sclerosis, based on their individual treatment needs,” Roche CMO Levi Garraway said in a statement.

The FDA’s approval is backed by pivotal data from the Phase III OCARINA II trial, which showed “no clinically significant” difference in levels of Ocrevus in the blood after subcutaneous administration. Ocrevus Zunovo’s safety profile was also consistent with that of the intravenous formulation.

September 13

Product: Eli Lilly’s Ebglyss

Indication: Atopic dermatitis (eczema)

Also on Friday, Eli Lilly secured approval for its therapeutic antibody Ebglyss to treat atopic dermatitis (eczema), after being denied in 2023 due to manufacturing concerns. Ebglyss, which is administered via a subcutaneous injection and can be given with or without topical corticosteroids, is authorized for adults and children 12 years and older who weigh at least 40 kg.

“Ebglyss offers a targeted approach to reduce a main cause of eczema inflammation,” Daniel Skovronsky, CSO and president of Lilly Research Laboratories, said in a statement, adding that that the treatment provides long-lasting symptom relief with a convenient once-monthly maintenance dose.

Lilly supported its application for Ebglyss with data from the Phase III ADvocate 1 and ADvocate 2 studies, in which 38% of patients on the treatment saw clear or almost-clear skin after 16 weeks. This was compared to the placebo arm, where only 12% of patients achieved this result.

Lilly plans to make Ebglyss available in the U.S. in the “coming weeks,” according to its announcement.

September 12

Product: Tecentriq Hybreza

Indication: Various cancers

The FDA has approved the first subcutaneous PD-(L)1 blocker in the form of Roche’s Tecentriq Hybreza, according to the company, which announced the news on Thursday.

Tecentriq Hybreza, which uses Halozyme Therapeutics’ Enhanze technology for subcutaneous administration, is indicated for all of the same cancers as its intravenous formulation, including several types of skin, liver, lung and soft-tissue cancers. The subcutaneous formulation “can treat patients faster and in more accessible settings,” Roche CMO Levi Garraway said in a statement.

Roche supported its application for Tecentriq Hybreza with data from the Phase Ib/III IMscin001 study, where the subcutaneous formulation yielded comparable levels of the drug in the blood to the intravenous version, with similar safety and efficacy profiles. Results from the Phase II IMscin002 study showed that 71% of patients preferred Hybreza to the intravenous formulation.

September 11

Product: Johnson & Johnson’s Tremfya

Indication: Ulcerative colitis

Patients with ulcerative colitis (UC) have a new treatment option as the FDA approved Johnson & Johnson’s Tremfya for the condition. According to J&J, Tremfya is the first and only fully-human, dual-acting monoclonal antibody for UC.

The approval is backed by results from the Phase IIb/III QUASAR study, in which participants given 200 mg of Tremfya every four weeks saw clinical remission at 44 weeks, compared with 19% in the placebo group.

This is the third indication for Tremfya, which is also approved to treat moderate-to-severe plaque psoriasis and psoriatic arthritis.

September 5

Product: Travere Therapeutics’ Filspari

Indication: IgA nephropathy

The FDA on Thursday granted full approval to Travere Therapeutics’ Filspari, closing the loop on an accelerated approval given in February 2023. Filspari is authorized for the treatment of patients with primary IgA nephropathy.

The traditional approval also expands Filspari’s indication, allowing for its use to slow the decline of kidney function in adult patients who are at risk of disease progression—while also removing a previous requirement of having a specific urinary protein level.

Accelerated approval for Filspari was granted based on data from the Phase III PROTECT study, which showed that the small molecule blocker lowered urine protein levels more than three times as much as the active control, Avapro. Serving also as the confirmatory trial, PROTECT narrowly missed the efficacy endpoint of the total slope of estimated glomerular filtration rate.

August 30

Product: Novavax’s COVID-19 vaccine

Indication: COVID-19

And Novavax makes three. On Friday, the FDA authorized the company’s updated COVID-19 vaccine as the third booster option for Americans this fall.

Like Moderna’s updated Spikevax and Pfizer/BioNTech’s updated Comirnaty, Novavax’s vaccine corresponds to the Omicron variant JN.1 strain of SARS-CoV-2 and “more closely targets” the currently circulating variants, helping to prevent hospitalizations and death, according to the FDA’s announcement.

The vaccine, which does not use mRNA technology but instead contains a recombinant version of a specific viral protein, as well as an adjuvant to boost immune protection, is approved under emergency use authorization.

August 29

Product: Emergent BioSolutions’ ACAM2000

Indication: Mpox

There is a second vaccine available in the battle against mpox after the FDA expanded its authorization for Emergent BioSolutions’ smallpox vaccine ACAM2000 to prevent infection in individuals deemed at high risk of the disease. ACAM2000, a live vaccinia virus vaccine that contains non-active components and cannot cause disease or be transmitted, joins Bavarian Nordic’s Jynneos on the market.

The FDA’s decision comes two weeks after the World Health Organization declared the mpox outbreak in Africa a public health emergency of international concern.

Prior to the approval, Emergent announced it would donate 50,000 doses of its vaccine to the humanitarian relief organization Direct Relief, which will deliver the shots to “difficult-to-reach locations,” in partnership with local and regional health authorities.

August 22

Product: Moderna’s Spikevax

Indication: COVID-19

The FDA is preparing for fall vaccination season, approving two updated COVID-19 vaccines, including Moderna’s Spikevax.

The new formulation of Moderna’s mRNA-based vaccine targets the Omicron KP.2 variant of the SARS-CoV-2 virus and is intended to help prevent COVID-19 in people six months and older. The new vaccine was fully approved for individuals 12 and older and approved under emergency use authorization for those six months to 11 years of age.

The new formulation, which was authorized based on a combination of manufacturing and preclinical data, as well as previous clinical, non-clinical and real-world evidence, is expected to be available in the U.S. in the coming days, according to Moderna’s press release.

August 22

Product: Pfizer and BioNTech’s Comirnaty

Indication: COVID-19

Also on Thursday, Pfizer and BioNTech announced FDA approval for their updated version of COVID-19 vaccine Comirnaty.

As with Spikevax, Pfizer and BioNTech’s vaccine is tailored to the KP.2 strain of the SARS-CoV-2 Omicron JN.1 lineage, based on guidance from the FDA. It is also fully approved for individuals 12 years and older and approved under emergency use authorization for those six months to 11 years of age.

The approval is based on “the full body of previous clinical, non-clinical, and real-world evidence supporting the safety and effectiveness,” of the companies’ COVID-19 vaccines, according to their joint press release. It is also expected to be available in the U.S. in the coming days.

August 15

Product: AstraZeneca’s Imfinzi

Indication: Perioperative non-small cell lung cancer

AstraZeneca won a new indication for Imfinzi on Thursday when the FDA green lit the drug, along with platinum-containing chemotherapy, for the perioperative treatment of non-small cell lung cancer (NSCLC). Under the expanded label, Imfinzi can be given with chemotherapy to NSCLC patients who have no known EGFR or ALK mutations. After surgery, Imfinzi can be given alone.

The approval came despite the FDA’s Oncologic Drugs Advisory Committee expressing skepticism about using Imfinzi both before and after surgery due to the risk of overtreatment.

AstraZeneca supported its supplemental application with data from the Phase III AEGEAN study, in which the Imfinzi regimen elicited a 17.21% pathological complete response rate, compared to 4.28% in the placebo arm. Event-free survival was also significantly better in Imfinzi-treated patients.

August 14

Product: Gilead’s Livdelzi

Indication: Primary biliary cholangitis

Gilead Sciences’ $4.3 billion acquisition of CymaBay paid dividends Wednesday after the FDA approved a key asset Livdelzi (seladelpar) to treat patients with primary biliary cholangitis (PBC), an autoimmune liver disease. Specifically, Livdelzi is approved alongside ursodeoxycholic acid (UDCA) in adult patients who cannot tolerate UDCA or who have otherwise shown an inadequate response to UDCA monotherapy.

Gilead Chairman and CEO Daniel O’Day called Livdelzi “an important new option” for patients and doctors, adding, “People living with PBC [primary biliary cholangitis] have been waiting for treatment advancements for many years.”

Livdelzi’s approval is supported by the Phase III RESPONSE trial, in which treatment with the drug elicited a significantly higher rate of composite biochemical response. After 12 months, 62% of patients in the Livdelzi arm met this composite endpoint versus 20% of placebo comparators.

August 14

Product: Incyte and Syndax’s Niktimvo

Indication: Chronic graft-versus-host disease

Incyte won its second chronic graft-versus-host disease (GVHD) FDA approval Wednesday as the regulator green lit Niktimvo, developed in partnership with Syndax Pharmaceuticals. Niktimvo is specifically intended for patients with chronic GVHD who have failed at least two prior lines of systemic therapy.

Niktimvo, which can be given to adult and pediatric patients weighing at least 40 kg, is the first FDA-approved anti-CSF-1R antibody directed against inflammatory and fibrotic drivers in GVHD, according to Incyte.

The approval was backed by the Phase II open-label AGAVE-201 trial, where treatment with a 0.3-mg/kg dose of Niktimvo elicited an overall response rate of 75%, with a median time to response of 1.5 months. By 12 months, 60% of patients were able to maintain a treatment response.

August 12

Product: Sandoz’s Enzeevu

Indication: Neovascular age-related macular degeneration

Sandoz bolstered its biosimilar position in the ophthalmology space Monday with an approval for Enzeevu, a 2-mg vial kit and prefilled syringe for intravitreal injection to treat neovascular age-related macular degeneration (nAMD). In addition, the FDA determined Enzeevu to be interchangeable with its reference medicine, Regeneron’s Eylea, and the drug holds unexpired exclusivity for first interchangeable biosimilar products.

Approval of Enzeevu was granted based on “the totality of evidence, including comprehensive analytical and preclinical in vitro study data,” in addition to clinical data from the Mylight study, which showed equivalent efficacy, safety and immunogenicity to Eylea, according to Sandoz’s press release.

August 12

Product: Ascendis Pharma’s Yorvipath

Indication: Hypoparathyroidism

After a long regulatory road, Ascendis Pharma received FDA approval Monday for Yorvipath (TransCon PTH) for the treatment of hypoparathyroidism, a rare endocrine disease characterized by abnormally low levels of the parathyroid hormone.

The FDA rejected Ascendis’ first attempt for approval of Yorvipath in May 2023, citing manufacturing concerns. For its second application, the FDA set a PDUFA date of May 14, 2024, but this was pushed back by three months after Ascendis filed a major amendment to the application.

According to Ascendis’ press release, Yorvipath is the first treatment for hypoparathyroidism in adults.

August 9

Product: Lykos Therapeutics’ Midomafetamine

Indication: Post-traumatic stress disorder

In one of the year’s most highly anticipated decisions, the FDA declined to approve Lykos Therapeutics’ midomafetamine (MDMA)-assisted therapy for post-traumatic stress disorder.

In its complete response letter (CRL), the regulator said the application “could not be approved based on data submitted to date” and requested another Phase III study to further assess the safety and efficacy of the treatment, according to Lykos. Lykos, which announced the CRL Friday evening, said it plans to request a meeting with the FDA to ask for reconsideration of the decision, and to get the agency’s recommendations for a resubmission.

The FDA’s decision follows a June advisory committee meeting in which the members voted 10-1 against recommending Lykos’ treatment.

August 9

Product: ARS Pharmaceuticals’ neffy

Indication: Severe allergic reactions

On Friday, the FDA approved ARS Pharmaceuticals’ neffy as the first epinephrine nasal spray for severe allergic reactions, providing a new option for 40 million Americans, according to ARS. With the approval, neffy becomes the only needle-free epinephrine medicine for the treatment of type I allergic reactions, including anaphylaxis, in adults and children who weigh at least 30 kg.

“Anaphylaxis is life-threatening and some people, particularly children, may delay or avoid treatment due to fear of injections,” Kelly Stone, associate director of the division of pulmonology, allergy and critical care in the FDA’s Center for Drug Evaluation and Research, said in a statement. “The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis.”

Neffy’s approval is based on four studies in 175 healthy adults, without anaphylaxis, that measured epinephrine concentrations in the blood following administration of the nasal spray or approved epinephrine injection products. Results of these studies showed comparable epinephrine blood concentrations between the two treatments.

August 8

Product: Citius Pharmaceuticals’ Lymphir

Indication: T cell lymphoma

Citius scored approval Thursday for Lymphir—the only treatment for cutaneous T cell lymphoma that targets the IL-2 receptor located on malignant T cells and Tregs.

Lymphir, Citius’ first FDA-approved product, is a reformulation of the FDA-approved cancer treatment denileukin diftitox, which combines the IL-2 cytokine and the diphtheria toxin. It works by binding to IL-2 receptors on malignant T cells and immunosuppressive regulatory T cells and releasing its toxin payload, causing the death of its target cells.

This was Citius’ second try for FDA approval, after the regulator rejected Lymphir in July 2023, outlining the need for “enhanced product resting requirements.” In its resubmitted data package, the company included findings from the from the Phase III Study 302 trial, where treatment with the drug led to an objective response rate of 36.2%, with 8.7% reaching complete response. Trial participants had already undergone a median of four prior anticancer therapies.

August 8

Product: Novartis’ Fabhalta

Indication: IgA nephropathy

Novartis racked up another indication for complement inhibitor Fabhalta Thursday, and in so doing, made history with the first such therapy for IgA nephropathy (IgAN). Fabhalta is also approved for paroxysmal nocturnal hemoglobinuria, a rare blood disease.

With Thursday’s accelerated approval, Fabhalta is now indicated for patients with IgAN who are at risk of “rapid disease progression,” according to Novartis, defined as patients with a urine protein-to-creatinine ratio of at least 1.5 g/g. The FDA’s decision was based on the Phase III APPLAUSE-IgAN study, in which treatment with Fabhalta cut proteinuria by 44% through nine months of follow-up, while placebo comparators saw only a 9% reduction in proteinuria.

August 7

Product: Amneal’s Crexont

Indication: Parkinson’s disease

Second time was the charm for Amneal Pharmaceuticals, which won approval Wednesday for Crexont, its novel oral formulation of carbidopa/levodopa (CD/LD), for Parkinson’s disease.

In the RISE-PD trial, treatment with Crexont elicited significantly longer Good On time, defined as the total time without dyskinesia, even when dosed less frequently than existing immediate-release CD/LD formulations.

The FDA previously rejected Crexont in June 2023, citing insufficient safety data, as reported by Reuters. Amneal resubmitted the application in February 2024 with additional data from a healthy volunteer study of the drug.

August 1

Product: Adaptimmune Therapeutics’ Tecelra

Indication: Synovial sarcoma

In one of the summer’s most highly anticipated decisions, the FDA approved Adaptimmune Therapeutics’ Tecelra as the first engineered T cell therapy for solid tumor cancers in the U.S. Tecelra is also the first new therapeutic option for synovial sarcoma in a more than 10 years, according to Adaptimmune.

Tecelra’s approval is backed by data from the SPEARHEAD-1 study, where treatment with the T cell therapy elicited an overall response rate of 43%. A complete response was reported in 4.5% of patients. Tecelra was tagged with a boxed warning for cytokine release syndrome (CRS), which could become severe or life-threatening, according to its label.

The approval is a “momentous step” toward the Adaptimmune’s goal to “redefine the way cancer is treated,” CEO Adrian Rawcliffe said in a statement Thursday.

August 1

Product: GSK’s Jemperli

Indication: Endometrial cancer

GSK racked up another indication Thursday for its blockbuster cancer therapy Jemperli. The PD-1–blocking antibody is now FDA approved in combination with carboplatin and the chemotherapy paclitaxel followed by Jemperli as a single agent to treat patients with primary advanced or recurrent endometrial cancer.

The approval also broadens Jemperli’s scope to include patients with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumors—a group that comprises 70% to 75% of endometrial cancer patients, according to GSK.

The approval is backed by data from Part 1 of the Phase III RUBY trial, where treatment with the Jemperli regimen led to a 31% reduction in risk of death compared to chemotherapy alone.

July 31

Product: Johnson & Johnson’s Darzalex Faspro

Indication: Multiple myeloma

Johnson & Johnson picked up another indication for its Darzalex Faspro Wednesday as the FDA approved the therapy, in combination with bortezomib, lenalidomide and dexamethasone, to treat newly diagnosed multiple myeloma patients who are eligible for autologous stem cell transplant. The quadruplet therapy “may significantly improve long-term outcomes,” according to J&J.

The approval is backed by data from the pivotal Phase III PERSEUS study, where treatment with subcutaneous Darzalex Faspro reduced the risk of disease progression or death by 60% in the intended patient population compared to standard of care. The overall safety profile for the therapy was consistent with the known safety profiles for Darzalex Faspro.

July 29

Product: Alpha Cognition’s Zunveyl

Indication: Alzheimer’s disease

There’s a new Alzheimer’s treatment on the market. Monday, the FDA gave the go-ahead to Alpha Cognition’s Zunveyl as just the second oral therapy approved this decade for Alzheimer’s disease.

Zunveyl was designed to eliminate drug absorption in the GI tract, potentially addressing tolerability issues with leading Alzheimer’s drugs, according to Alpha Cognition. A prodrug of galantamine and an acetylcholinesterase inhibitor, Zunveyl is thought to work by preventing the breakdown of acetylcholine, a neurotransmitter involved in memory, motivation and attention functions.

Zunveyl’s approval is backed by chemistry, manufacturing and controls information and data demonstrating its bioequivalence and tolerability compared to galantamine immediate-release tablets and galantamine extended-release capsules. Minimal adverse events were reported in these trials, according to the company’s press release.

July 29

Product: Grifols’ Xembify

Indication: Primary humoral immunodeficiencies

Treatment-naïve patients with primary humoral immunodeficiencies can now access Grifols’ Xembify after the FDA approved an expanded label for the subcutaneous immunoglobulin treatment (SCIg). Patients will now be eligible to begin treatment with Xembify without initial intravenous administration. With the approval, Xembify becomes the first 20% SCIg with an extended label for treatment-naïve patients, according to its maker.

The FDA has additionally greenlit biweekly dosing of the SCIg therapy, backed by a Phase IV trial that showed non-inferiority in total Ig levels compared to weekly administration. Grifols intends to launch the new label this quarter.

This is the second win for Grifols in as many months, after the FDA approved Yimmugo, an intravenous immunoglobulin (Ig) therapeutic, to treat primary immunodeficiencies developed by its subsidiary Biotest.

July 25

Product: Sun Pharma’s Leqselvi

Indication: Alopecia Areata

Adult patients with severe alopecia areata, an autoimmune disease that leads to hair loss, have a new treatment option after the FDA approved Sun Pharma’s Leqselvi on Thursday. An oral JAK inhibitor, Leqselvi interrupts the pathways thought to contribute to hair loss in severe alopecia areata, according to Sun.

The approval is supported by two randomized, double-blind, placebo-controlled Phase III trials that enrolled a total 1,220 patients with alopecia areata who had at least 50% scalp hair loss. At baseline, the average trial participant had 13% scalp coverage. After 24 weeks of treatment with Leqselvi, more than 30% of patients experienced 80% or more scalp coverage; up to 25% of patients had almost all of their scalp hair back after 24 weeks, Sun reported.

July 24

Product: BioMarin’s Brineura

Indication: Batten disease

BioMarin secured an expanded approval Wednesday for Brineura, its treatment for the genetic condition called neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The treatment, which was previously indicated only for symptomatic children 3 years of age and older with late infantile CLN2 disease, also known as Batten disease, can now be given to diagnosed children of all ages, regardless of whether they are symptomatic or presymptomatic.

“Today’s approval represents a significant step forward in enabling children to be treated with Brineura as early as possible, when we can have the greatest impact in altering the natural course of disease,” Hank Fuchs, president of worldwide research and development at BioMarin, said in a statement.

The label expansion is based on a Phase II trial that evaluated Brineura in children aged 1–6 over three years, with eight patients being less than 3 years old. The results showed that Brineura slowed the decline in motor function and delayed disease onset, according to BioMarin. The safety results in children under 3 years was similar to the therapy’s known safety profile.

July 18

Product: Phathom Pharmaceuticals’ Voquezna

Indication: Non-erosive gastroesophageal reflux disease

Phathom Pharmaceuticals secured a third indication for its acid suppression drug Voquezna on Thursday. Previously approved to treat H. pylori infection and erosive gastroesophageal reflux disease (GERD), Voquezna is now authorized for the non-erosive form of the disease.

The non-erosive GERD approval was supported by a pivotal trial in which Voquezna quickly and significantly reduced heartburn with daily treatment compared to placebo over four weeks. The median percentage of heartburn-free days was 48% for patients treated with Phathom’s drug, compared to 17% for placebo-treated participants.

The approval significantly opens the market opportunity for Voquezna, as an estimated 15 million people in the U.S. are treated with prescription drugs for non-erosive GERD compared to approximately 7 million for erosive GERD.

July 16

Product: Orexo’s OX124

Indication: Opioid overdose

Second time is not the charm for Orexo, as the FDA denied its application for OX124, a high-dose naloxone nasal spray for the rescue treatment for opioid overdose.

In its Complete Response Letter, the FDA requested another “human factors” study before the company files another application. The request is aligned with previous communication between Orexo and the FDA, the company noted in its press release, and Orexo has already completed such a study. The CRL also requested additional technical data on the final commercial product, which Orexo said was “unexpected.”

The FDA previously rejected OX124 in April 2023, citing “technical issues with the equipment used for the secondary packaging process.”

July 10

Product: Novo Nordisk’s insulin icodec

Indication: Diabetes mellitus

Novo Nordisk was hit with a rejection Wednesday for its once-weekly basal insulin icodec injection for the treatment of diabetes mellitus. It appears Novo was tripped up by manufacturing issues, as the company said the FDA made requests related to the manufacturing process in its Complete Response Letter. The regulator also raised concerns related to the use of insulin icodec in type 1 diabetes (T1D). Novo does not expect to be able to address the requests this year.

The FDA’s decision follows an advisory committee vote that went against Novo’s application. In a 7-4 vote, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee agreed the current evidence shows icodec’s benefits do not outweigh its risks. The advisers specifically pointed to the heightened risk of hypoglycemia versus the once-daily Tresiba (insulin degludec) in T1D patients.

July 9

Product: Arcutis Biotherapeutics’ Zoryve

Indication: Atopic dermatitis

Arcutis Biotherapeutics won its third indication in two years for Zoryve, as the FDA green lit the topical cream to treat adults and children six years and older with atopic dermatitis. Arcutis said Zoryve will be available through wholesalers and dermatology pharmacies by the end of July.

The approval is supported by three Phase III trials, including INTEGUMENT-1 and INTEGUMENT-2. Both studies met the primary endpoint of treatment success as determined by a score of clear or almost clear on the validated Investigator Global Assessment-Atopic Dermatitis scale, alongside a two-grade improvement at four weeks.

Zoryve is also approved to treat patients six years and older with plaque psoriasis and seborrheic dermatitis in people nine years and older.

July 2

In one of the year’s most highly anticipated decisions, the FDA on Tuesday approved Eli Lilly’s donanemab, an anti-amyloid antibody designed to modify the course of Alzheimer’s disease. Donanemab, which will be marketed as Kisunla, will compete with Biogen and Eisai’s Leqembi, greenlit last year, which brought in $19 million for its makers in Q1 2024.

The FDA’s decision comes less than a month after an advisory committee voted unanimously in donanemab’s favor. This was despite some concerns outlined in briefing documents regarding Lilly’s trial methodology, including its use of the integrated Alzheimer’s Disease Rating Scale (iADRS) as the primary endpoint, the use of tau protein levels to decide study inclusion and the cessation of donanemab treatment in patients whose brain amyloid levels dropped past a certain, pre-specified threshold.

June 28

Product: Rocket Pharmaceuticals’ Kresladi

Indication: Severe leukocyte adhesion deficiency-I (LAD-I)

Rocket Pharmaceuticals announced Friday that the FDA rejected its rare disease gene therapy Kresladi (marnetegragene autotemcel) in a Complete Response Letter requesting “limited additional” chemistry, manufacturing and controls information to complete the regulator’s review.

Kresladi, a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I), has already faced delays from the agency as it was initially supposed to find out its fate in March 2024. However, the FDA pushed back the PDUFA date to June 30.

Rocket said it has met with leaders from the FDA’s Center for Biologics Evaluation and Research (CBER) to “align on the limited scope” of additional chemistry, manufacturing and controls (CMC) information and support Kresladi’s approval as soon as possible. However, Rocket’s stock price fell by over 11% in premarket trading Friday.

June 27

Product: Merck and Daiichi Sankyo’s patritumab deruxtecan

Indication: Non-small cell lung cancer

A highly anticipated decision went against Merck and Daiichi Sankyo after the FDA cited issues with a third-party manufacturing facility in relation to their Biologics License Application for patritumab deruxtecan in non-small cell lung cancer (NSCLC). The drug development partners had been seeking approval for patritumab deruxtecan, an antibody-drug conjugate (ADC), to treat patients with advanced or metastatic NSCLC bearing certain genetic mutations.

In its Complete Response Letter, the FDA did not identify efficacy or safety problems with the application, according to the companies. Merck and Daiichi Sankyo will “work closely with the FDA and the third-party manufacturer to address the feedback as quickly as possible,” Ken Takeshita, global head of R&D at Daiichi Sankyo, said in a statement.

June 26

Product: Verona’s Ohtuvayre

Indication: Chronic obstructive pulmonary disease

Patients with chronic obstructive pulmonary disease (COPD) have their first inhaled treatment with a new mechanism of action in more than 20 years after the FDA greenlit Verona Pharma’s Ohtuvayre. A selective dual inhibitor of the PDE3 and PDE4 enzymes, Ohtuvayre produces both bronchodilator and non-steroidal anti-inflammatory effects.

Wednesday’s approval is supported by data from the Phase III ENHANCE-1 and ENHANCE-2 studies, where Ohtuvayre significantly improved lung function in COPD patients. The drug also significantly reduced the rate of moderate or severe exacerbations, while prolonging the time before patients experienced their first exacerbation episode.

Ohtuvayre—which Verona plans to launch in the third quarter of this year—will enter a U.S. market of over 16 million potential patients.

June 26

Product: AbbVie and Genmab’s Epkinly

Indication: Follicular lymphoma

AbbVie and Genmab’s bispecific antibody Epkinly can now be used to treat patients with relapsed or refractory follicular lymphoma. The FDA approval makes Epkinly the first T cell-engaging bispecific antibody for the subcutaneous treatment of relapsed or refractory follicular lymphoma, according to the partners.

AbbVie and Genmab made their case for Epkinly’s approval with data from the Phase I/II EPCORE NHL-1 trial, where the drug elicited an objective response rate of 82%, including a 60% complete response rate and a 22% partial response rate. The trial included 127 adult follicular lymphoma patients who had undergone a median of three prior lines of therapy.

Epkinly was first approved in May 2023 for relapsed or refractory diffuse large B-cell lymphoma.

June 25

Product: AbbVie’s ABBV-951

Indication: Parkinson’s disease

For the second time in as many years, the FDA declined to approve AbbVie’s experimental Parkinson’s treatment ABBV-951, citing issues with a third-party manufacturer.

This appears to be the main issue, as the FDA’s Complete Response Letter did not reveal any issues with the safety or efficacy of ABBV-951 or its administration device, according to AbbVie. The regulator is also not requesting that AbbVie conduct additional efficacy and safety trials related to the drug or device-related testing.

ABBV-951—a solution of two prodrugs, cabidopa and levodopa—is approved in over 34 other countries.

June 22

Product: Bristol Myers Squibb’s Krazati

Indication: Colorectal cancer

Bristol Myers Squibb’s oncology franchise is celebrating another approval after the FDA greenlit Krazati in combination with cetuximab for patients with previously treated KRASG12C-mutated locally advanced or metastatic colorectal cancer. The approval is the second for Krazati, which is also FDA-authorized to treat KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer.

The new approval reinforces “Krazati’s potential across tumor types,” the company said in a press release.

In the Phase I/II KRYSTAL-1 study, Krazati plus cetuximab elicited a confirmed overall response rate of 34%, all of which were partial responses, meeting the primary endpoint, according to BMS. The median duration of response was 5.8 months.

June 21

Product: argenx’s Vyvgart Hytrulo

Indication: Chronic inflammatory demyelinating polyneuropathy

Patients with chronic inflammatory demyelinating polyneuropathy (CIDP) have a new treatment option after the FDA greenlit argenx’s Vyvgart Hytrulo as the first neonatal Fc receptor (FcRn) blocker for the condition. Vyvgart Hytrulo represents the first novel, precision mechanism of action in more than 30 years for CIDP, a rare, debilitating neuromuscular disorder of the peripheral nervous system, according to argenx’s press release.

The approval is based on the ADHERE study, where 69% of patients treated with Vyvgart Hytrulo regardless of prior treatment saw evidence of clinical improvement, including improvements in mobility, function and strength.

Vyvgart Hytrulo, which is also approved in the U.S. for the treatment of generalized myasthenia gravis, is available as a once weekly 30-to-90 second subcutaneous injection.

June 20

Product: Sarepta’s Elevidys

Indication: Duchenne muscular dystrophy

Sarepta Therapeutics secured a much-anticipated expanded approval Thursday for Elevidys, its gene therapy for Duchenne muscular dystrophy (DMD). Elevidys—which was previously approved only for 4 - to 5-year-olds who could walk—will now be available to patients who are at least 4 years old with a confirmed mutation in the DMD gene, regardless of ambulatory status.

The FDA granted full approval to all patients aged 4 and older with a confirmed mutation in the DMD gene, while giving accelerated approval to non-ambulatory patients.

The decision comes despite the gene therapy missing the primary endpoint in its Phase III confirmatory study. In its decision, the FDA said the review of the secondary and exploratory endpoints was “compelling” enough to show a clinical benefit.

June 18

Product: AbbVie’s Skyrizi

Indication: Ulcerative colitis

AbbVie’s blockbuster therapy Skyrizi now has further reach after the FDA greenlit the IL-23 antagonist for the treatment of moderately to severely active ulcerative colitis. Skyrizi is the first drug of its class approved for both ulcerative colitis and Crohn’s disease, according to the company’s announcement.

The label expansion was supported by two Phase III trials—a 12-week induction study and a 52-week maintenance study—both of which met the primary endpoint of clinical remission. The studies also met a key secondary endpoint, endoscopic improvement.

The approval marks for the fourth indication for Skyrizi, which is also FDA-authorized to treat plaque psoriasis and psoriatic arthritis.

June 17

Product: Merck’s Capvaxive

Indication: Invasive pneumococcal disease and pneumococcal pneumonia

The FDA has approved the first pneumococcal vaccine designed for adults. Merck’s Capvaxive is formulated to cover pneumococcal strains responsible for approximately 84% of invasive pneumococcal disease.

The vaccine’s accelerated approval is based partly on the Phase III STRIDE-3 study, in which Capvaxive was non-inferior to a commercially available 20-valent comparator vaccine. Merck’s shot also elicited superior immune responses for 10 of the 11 serotypes that were not covered by the comparator.

“Capvaxive is designed to include the serotypes that cause the majority of invasive pneumococcal disease in adults,” Walter Orenstein, professor emeritus of medicine, epidemiology, global health and pediatrics at Emory University who serves on Merck’s scientific advisory committee, said in a company statement. “Many cases of adult disease are caused by serotypes not included in other approved pneumococcal conjugate vaccines.”

June 17

Product: Merck’s Keytruda

Indication: Endometrial carcinoma

Merck is celebrating a milestone for Keytruda after the FDA greenlit the blockbuster immunotherapy, in combination with chemotherapy, to treat primary advanced or recurrent endometrial carcinoma. The approval marks the 40th U.S. indication for Keytruda, and its third in this type of cancer.

Specifically, Keytruda is approved in combination with carboplatin and paclitaxel, followed by the anti-PD-1 therapy, as a single agent. The approval is supported by data from the Phase III NRG-GY018/ KEYNOTE-868 trial, in which the regimen reduced the risk of disease progression or death by 40% in patients whose cancer was mismatch repair proficient and by 70% in patients whose cancer was mismatch repair deficient, when compared to placebo with carboplatin and paclitaxel followed by placebo alone.

June 17

Product: AstraZeneca’s Imfinzi

Indication: Endometrial cancer

Monday, the FDA granted approval for AstraZeneca’s Imfinzi, in combination with chemotherapy, to treat primary advanced or recurrent endometrial cancer that is mismatch repair (MMR) deficient.

The approval was backed by results of a prespecified exploratory subgroup analysis by MMR status in the DUO-E Phase III trial where Imfinzi plus chemotherapy followed by Imfinzi monotherapy reduced the risk of disease progression or death by 58% versus chemo alone.

“Imunotherapy in combination with chemotherapy is emerging as a new standard of care in this setting, and the approval of Imfinzi offers an important new option for patients with mismatch repair deficient disease,” Dave Fredrickson, executive vice president of the oncology business unit at AstraZeneca, said in a press release.

June 17

Product: Biotest’s Yimmugo

Indication: Primary immunodeficiencies

Grifols subsidiary Biotest got the FDA’s green light for its first U.S.-approved medicine, Yimmugo, an intravenous immunoglobulin (Ig) therapeutic, to treat primary immunodeficiencies.

Yimmugo is the first of a “threesome of Biotest plasma proteins on the horizon destined for markets including the U.S.,” according to Grifols’ press release, which also includes a fibrinogen concentrate for acquired fibrinogen deficiency and a polyvalent immunoglobulin to treat community-acquired pneumonia or severe community-acquired pneumonia.

Grifols and Biotest plan to launch Yimmugo in the U.S. in the second half of 2024.

June 14

Product: Amgen’s Blincyto

Indication: B-ALL

Patients with an aggressive blood cancer have a new treatment option after the FDA approved Amgen’s Blincyto for CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase.

“Today’s approval in the frontline consolidation phase, regardless of MRD status, allows us to reach more patients than ever with this transformative, first-in-class Bispecific T-cell Engager (BiTE®) therapy,” Jay Bradner, executive vice president of research and development and chief scientific officer at Amgen, said in a press release.

The approval, a third indication for Blincyto, was supported by the Phase III E1910 clinical trial, in which Amgen’s drug plus multiphase consolidation chemotherapy showed superior overall survival versus chemotherapy alone.

June 13

Product: BMS’s Augtyro

Indication: NTRK-positive, locally advanced or metastatic solid tumors

Thursday, the FDA granted accelerated approval to Bristol Myers Squibb’s tyrosine kinase inhibitor (TKI) Augtyro for the treatment of NTRK-positive, locally advanced or metastatic solid tumors. Augtyro can now be used in patients aged 12 years and older whose disease has progressed after initial treatment as well as patients with no satisfactory alternative therapies and those who are likely to suffer from “severe morbidity” due to surgical resection, according to BMS.

The approval is supported by data from the Phase I/II TRIDENT-1 trial, where treatment with Augtyro resulted in a 58% confirmed objective response rate (cORR) in TKI-naïve patients, of which 15% were complete responses. At the time of analysis, median duration of response in this subgroup had not been reached. In TKI-pretreated patients, BMS’ drug elicited a 50% cORR, with a median response duration of 9.9 months.

Augtyro was first approved in November 2023 for ROS1-positive advanced or metastatic non-small cell lung cancer.

June 10

Product: Ipsen and Genfit’s Iqirvo

Indication: Primary biliary cholangitis

Monday, the FDA approved the first new drug in nearly a decade for primary biliary cholangitis: Ipsen and Genfit’s Iqirvo. A rare liver disease, PBC affects around 100,000 people in the U.S. and can lead to liver failure.

Iqirvo is intended to be used in combination with ursodeoxycholic acid (UDCA) in adult patients who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

The companies won accelerated approval for Iqirvo based on a reduction of alkaline phosphatase, a biochemical marker often used as a surrogate endpoint in PBC studies. Treatment with the drug demonstrated “statistically significant improvements in biochemical response compared to UDCA alone,” Christelle Huguet, executive vice president and head of research and development at Ipsen, said in a press release. An improvement in survival or prevention of liver decompensation events has not yet been shown, and the companies may need to run a confirmatory trial to verify Iqirvo’s clinical benefit.

June 10

Product: Almirall’s Klisyri

Indication: Actinic keratosis

Dermatology company Almirall secured expanded approval of Klisyri for larger actinic keratosis-affected areas of the face or scalp. Klisyri can now be used to treat lesions up to 100 cm² caused by the pre-cancerous dermatological condition, after safety and tolerability profiles were consistent with original pivotal trial results.

The new authorization for Klisyri, a microtubule inhibitor ointment, increases dosing for surface area treatment from up to 25 cm² to up to 100 cm², according to the company’s press release.

In the same press release, Almirall Chief Scientific Officer Karl Ziegelbauer called the expanded approval “a significant step forward for both patients and treating dermatologists,” adding that the latter “are looking for ways to treat the entire affected area to help prevent further lesion progression.”

June 7

Product: Geron’s Rytelo

Indication: Myelodysplastic syndromes

Geron Corporation kicked off the weekend on a high note as the FDA approval of its telomerase inhibitor Rytelo for myelodysplastic syndromes (MDS)—a group of blood cancers—sent the company’s stock soaring more than 30%. Rytelo is specifically approved for MDS patients with transfusion-dependent anemia who do not respond to or are ineligible for the standard-of-care treatment, erythropoiesis-stimulating agents.

The approval—Geron’s first after 34 years in business—was supported by data from the Phase III IMerge trial, in which patients on Rytelo had significantly higher rates of red blood cell transfusion independence over placebo for at least 24 weeks—28% in the treatment arm versus 3% on placebo. For those who responded, this was sustained for a median of 1.5 years.

June 7

Product: GSK’s Arexvy

Indication: Respiratory syncytial virus

People ages 50–59 at an increased risk of severe outcomes from respiratory syncytial virus have a new preventative option after the FDA greenlit GSK’s RSV vaccine Arexvy for this subgroup on Friday. Arexvy is indicated for the prevention of lower respiratory tract disease associated with RSV.

Friday’s label expansion—which was backed by strong immunogenicity and safety data in this population—extends the market reach for Arexvy, which became the first vaccine for RSV in May 2023, at that point intended for adults 60 and above.

GSK is also evaluating the vaccine for use in people 18-49 at increased risk of severe disease, and immunocompromised patients 18 and older.

May 31

Product: Moderna’s mRESVIA

Indication: Respiratory syncytial virus

Moderna has a second product on the market after the FDA approved mRESVIA—formerly mRNA-1345—to protect adults 60 years and older from respiratory syncytial virus (RSV). In a press release, Moderna CEO Stéphane Bancel touted “the strength and versatility” of the company’s mRNA platform, adding that the approval also marks the first time an mRNA vaccine has been approved for a disease other than COVID-19.

mRESVIA won approval based on the Phase III ConquerRSV trial, a global study of around 37,000 adults aged 60 or older in 22 countries, in which it displayed an efficacy rate of 83.7% against RSV lower respiratory tract disease. No serious safety concerns were identified in the trial.

May 30

Product: BMS’s Breyanzi

Indication: Mantle Cell Lymphoma

After winning approval earlier this month in follicular lymphoma, Bristol Myers Squibb’s Breyanzi got the FDA nod for another indication on Thursday: relapsed or refractory mantle cell lymphoma (MCL). Specifically, Breyanzi is approved for patients with MCL who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor.

The approval is backed by the results of the MCL cohort of TRANSCEND NHL 001, where treatment with Breyanzi elicited a 67.6% complete response rate in the target patient population.

Thursday’s approval marks the fourth indication for Breyanzi, “making it the CAR T cell therapy available to treat the broadest array of B-cell malignancies,” according to BMS’s press release.

May 29

Product: Eli Lilly’s Retevmo

Indication: RET-altered pediatric cancers

Eli Lilly won accelerated approval Wednesday for Retevmo to treat pediatric patients two years and older with RET-positive thyroid cancers and other solid tumors that carry the mutation. Retevmo is the first drug in the class available for children under 12 years of age, Pharmaphorum reported.

Retevmo is specifically indicated for advanced or metastatic medullary thyroid cancer with a RET mutation, advanced or metastatic thyroid cancer with a RET gene fusion untreatable with radioactive iodine therapy, and locally advanced or metastatic solid tumors with a RET gene fusion that have progressed after prior systemic treatment or have no treatment options, according to the publication.

The new approval for Retevmo, which was previously authorized to treat patients 12 and older with RET-positive thyroid cancers, is based on a single-arm study that showed an overall response rate of 48%, with a median duration of response not reached after 12 months of follow-up.

May 29

Product: Tris Pharma’s Onyda XR

Indication: Attention deficit hyperactivity disorder

Wednesday, the FDA greenlit Tris Pharma’s Onyda XR as the first non-stimulant medication for attention deficit hyperactivity disorder (ADHD) with a liquid formulation and nighttime dosing, according to the company. Onyda XR is a reformulation of clonidine hydrochloride, which was first approved by the FDA in 1974 to treat high blood pressure. Clonidine was approved for ADHD in 2010 under the brand name Kapvay, which is owned by Shionogi.

Onyda XR leverages Tris’ LiquiXR platform, producing a “smooth, extended-release profile,” per the biotech.

Approved for patients six years and older, Tris expects to have Onyda XR available in U.S. pharmacies by the second half of 2024.

May 29

Product: Teva’s Austedo XR

Indication: Tardive dyskinesia and Huntington’s disease chorea

People with tardive dyskinesia and Huntington’s disease chorea have a streamlined treatment option after the FDA approved a new one-pill-a-day version of Teva’s Austedo XR. The newly approved formulation “offers more flexibility with the most once-daily doses of any vesicular monoamine transporter 2 (VMAT2) inhibitor,” for these conditions, according to Teva’s press release. Austedo XR comes in four tablet strengths: 30, 36, 42 and 48 mg.

Austedo XR, a once-daily extended-release formulation, was first approved in February 2023.

May 28

Product: Amgen’s Bkemv

Indication: Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome

AstraZeneca’s rare disease drug Soliris now has a biosimilar on the market after the FDA greenlit Amgen’s Bkemv to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Bkemv was granted the FDA’s interchangeability designation, which allows it to be used in place of the branded reference product without needing to change the prescription.

Like Soliris, Bkemv carries a boxed warning for meningococcal infections, which according to its label can be “serious and life-threatening.” Thus, it is only available through a restricted Risk Evaluation and Mitigation Strategies program.

May 16

Product: Amgen’s Imdelltra

Indication: Small cell lung cancer

Amgen secured approval Thursday for its first-in-class bi-specific T-cell engager, Imdelltra, for extensive-stage small cell lung cancer (SCLC). With the FDA nod, Imdelltra becomes the first bispecific T-cell engager therapy for advanced SCLC.

The accelerated approval was based on a Phase II study of 99 patients in the target population, where Imdelltra led to an overall response rate of 40% and a median duration of response of 9.7 months. Imdelltra’s label contains a boxed warning for serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome, according to the FDA’s press release.

May 15

Product: BMS’s Breyanzi

Indication: Follicular lymphoma

Bristol Myers Squibb’s Breyanzi is now approved for the treatment of relapsed or refractory follicular lymphoma after the FDA granted a label expansion under its accelerated approval pathway. The approval was backed by data from the Phase II TRANSCEND FL study in which treatment with the CAR T cell therapy led to a 95.7% overall response rate, with a complete response rate of 73.4%.

Breyanzi, which first won approval in February 2021 for relapsed or refractory large B cell lymphoma, is also authorized to treat small lymphocytic leukemia and chronic lymphocytic leukemia. By May 31, the FDA is expected to decide whether to grant approval for the therapy in refractory mantle cell lymphoma.

May 14

Product: Dynavax’s Heplisav-B

Indication: Hepatitis B patients undergoing hemodialysis

The FDA declined to approve the supplemental Biologics License Application for Dynavax Technologies’ hepatitis B vaccine in patients undergoing hemodialysis, deeming the safety and efficacy data submitted by the company insufficient.

In its Complete Response Letter, the regulator said the data was insufficient because a third-party clinical site operator destroyed data source documents for about half of the subjects enrolled in the vaccine’s trial, according to Reuters.

While the vaccine, Heplisav-B, initially won approval for the prevention of hepatitis B in 2017, its path to the market was rocky, with two previous rejections in 2013 and 2016 for “unresolved safety concerns,” per Reuters.

May 1

Product: Boehringer Ingelheim’s Cyltezo

Indication: Rheumatoid arthritis, Crohn’s disease, ulcerative colitis and more

There’s another new biosimilar option to AbbVie’s blockbuster antirheumatic Humira. Wednesday, the FDA greenlit a high-concentration and citrate-free version of Boehringer Ingelheim’s Cyltezo, which was originally approved in October 2021. The newly approved dose is 100 mg/mL and is sold at a 5% discount to the branded reference product.

Cyltezo is indicated for all the same conditions as Humira, including moderate-to-severe rheumatoid arthritis, Crohn’s disease and ulcerative colitis. Wednesday’s approval is backed by data from the Phase I VOLTAIRE-HCLF study, which compared the bioavailability of the high- and low-concentration (50 mg/mL) formulation of Cyltezo in 200 healthy volunteers.

April 30

Product: Neurocrine Biosciences’ Ingrezza

Indication: Huntington’s disease

A more convenient version of Neurocrine Biosciences’ Ingrezza will be hitting the market to treat tardive dyskinesia and chorea in Huntington’s disease after the FDA closed out April by approving a sprinkle capsule formulation of the drug.

Like the original capsule version, which was approved in 2017 for tardive dyskinesia and in 2023 for chorea in Huntington’s, Ingrezza’s sprinkle formulation comes in 40-mg, 60-mg and 80-mg doses but is designed to be opened and sprinkled on soft foods. This format could be more accessible for patients who have trouble swallowing whole capsules, according to the Neurocrine’s announcement, which also noted that a survey of Huntington’s patients with chorea and their caregivers showed that 62% had difficulty swallowing due to their involuntary movements.

April 29

Product: Pfizer and Genmab’s Tivdak

Indication: Cervical cancer

The FDA has converted the accelerated approval of Pfizer and Genmab’s Tivdak into a full nod for recurrent or metastatic cervical cancer that has progressed on or after chemotherapy.

The antibody-drug conjugate (ADC), which was originally developed under a partnership between Seagen and Genmab, was granted accelerated approval in September 2021 based on a 24% objective response rate seen in the Phase II innovaTV 204 trial.

In the Phase III innovaTV 301 study, which enrolled more than 500 patients, Tivdak significantly boosted survival versus chemotherapy. An October 2023 readout showed the ADC cut the risk of death by 30% in patients with recurrent or metastatic cervical cancer; it also reduced the risk of death or worsening disease by 33% versus chemotherapy. No new safety signals were observed.

April 29

Product: X4 Pharmaceuticals’ Xolremdi

Indication: WHIM Syndrome

The FDA approved X4 Pharmaceuticals’ Xolremdi Monday as the first targeted treatment for WHIM syndrome, an ultra-rare immunodeficiency disease named for its four characteristics: warts, hypogammaglobulinemia, infections and myelokathexis.

Myelokathexis is a congential disorder of the white blood cells, and Xolremdi, an oral CXCR4 antagonist, is designed to mobilize white blood cells such as neutrophils, lymphocytes and monocytes from the bone marrow into the blood to improve immune deficiencies.

In the Phase III 4WHIM trial, Xolremdi showed a 60% reduction in annualized infection rate compared to placebo; trial participants had less than one infection per year compared with 4.5 for the placebo group. Patients saw an even greater reduction with additional time on treatment.

“It’s an exciting time for personalized medicine, and I think WHIM is going to be a poster child for rare diseases and the ability where we’re at now in modern medicine to design therapies to treat underlying genetic disorders,” Teresa Tarrant, an associate professor at Duke University’s School of Medicine and lead investigator of the 4WHIM trial, told BioSpace prior to Xolremdi’s approval.

April 26

Product: Pfizer’s Beqvez

Indication: Hemophilia B

People with moderate to severe hemophilia B have a new, one-time treatment option Friday, as the FDA approved Pfizer’s Beqvez.

The gene therapy is specifically approved for those patients who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to the adeno-associated virus serotype Rh74var (AAVRh74var) capsid. Beqvez is designed to help patients produce FIX themselves.

A one-time dose of Beqvez reduced bleeds post-treatment compared to standard of care with a median of zero bleeds after up to three years of follow-up, Pfizer reported, adding that this could potentially help patients avoid years of treatment burden. The current standard of care involves regular intravenous infusions of FIX, often administered multiple times a week or multiple times a month, according to the company.

Beqvez follows CSL Behring’s gene therapy Hemgenix, which became the first FDA-approved gene therapy for hemophilia B in November 2022. Pfizer’s first marketed gene therapy, Beqvez was approved by Health Canada in January, and was featured in BioSpace’s 5 Cell and Gene Therapy Decisions to Watch in 2024.

April 23

Product: Day One Biopharmaceuticals’ Ojemda

Indication: Pediatric low-grade glioma

The FDA has approved the first medicine for children with relapsed or refractory pediatric low-grade glioma (pLGG) with BRAF fusions or rearrangements, Day One Biopharmaceuticals announced Tuesday. The drug, Ojemda, is also the only systemic treatment option for pLGG that allows for a once-weekly treatment schedule, according to the company.

A type II RAF kinase blocker, Ojemda works by binding a key player in the MAPK signaling cascade, disrupting the tumor cells’ unchecked growth and proliferation and triggering cell death. In the first arm of the Phase II FIREFLY-1 study, treatment with Ojemda elicited a best overall response rate of 51%, which included 28% partial responses. The median duration of response was 13.8 months as of the data cut-off in June 2023.

Ojemda, which was approved under the FDA’s accelerated approval pathway, is being developed in partnership with XOMA Corporation.

April 23

Product: Amneal Pharmaceuticals’ OTC Naloxone hydrochloride nasal spray

Indication: Opioid overdose

There is now a generic alternative to over-the-counter NARCAN for the emergency treatment of opioid overdose after the FDA approved Amneal’s Naloxone Hydrochloride Nasal Spray on Tuesday.

“With today’s launch, Amneal is proud to help address this public health emergency by providing naloxone nasal spray at an affordable price and without a prescription,” co-CEOs Chirag and Chintu Patel said in a statement.

The nasal spray, which is manufactured in the U.S., is available in a 4 mg dose.

April 22

Product: Abeona Therapeutics’ prademagene zamikeracel

Indication: Recessive dystrophic epidermolysis bullosa

Chemistry, manufacturing and controls (CMC) issues led the FDA to reject Abeona Therapeutics’ investigational gene-corrected cell therapy prademagene zamikeracel (pz-cel) in recessive dystrophic epidermolysis bullosa, a rare disorder that causes painful blistering and erosion of the skin.

In its Complete Response Letter (CRL), the regulator highlighted specific CMC issues that “must be satisfactorily resolved” before the application can be approved. In particular, the FDA is looking for additional information regarding certain manufacturing and release testing methods. There were no issues with clinical or efficacy data and the FDA is not seeking additional trials or data to support pz-cel’s application, according to Abeona’s announcement.

In a statement, Abeona CEO Vish Seshadri expressed surprise and disappointment in the decision and said the company anticipates completing a BLA resubmission in the third quarter of 2024 “with necessary updates to fully satisfy all the deficiencies outlined in the CRL.”

Pz-cel was highlighted this week in BioSpace’s 5 Cell and Gene Therapy Decisions to Watch in 2024.

April 22

Product: ImmunityBio’s Anktiva

Indication: Non-muscle invasive bladder cancer

A large number of bladder cancer patients will have a new treatment option as the FDA on Monday greenlit ImmunityBio’s Anktiva, a first-in-class IL-15 superagonist for non-muscle invasive bladder cancer—which accounts for approximately 80% of new cases, according to the company. Anktiva is to be used in BCG-unresponsive patients with carcinoma in situ. It is to be used alongside the Bacillus Calmette-Guérin (BCG) vaccine, traditionally used to lower risk of tuberculosis and other mycobacterial infections but long recognized to induce an immune response in the bladder that can help fight off the cancer.

The approval is a long time coming for ImmunityBio after the FDA rejected Anktiva’s Biologics License Application in May 2023 due to issues with a pre-license inspection of a third-party manufacturer.

The company backed its application with results from the Phase II/III QUILT-3.032 study, in which maintenance treatment with Anktiva plus BCG elicited a complete response rate of 62%, with duration of response exceeding 47 months at the November 2023 cut-off.

April 18

Product: Genentech’s Alecensa

Indication: Non-small cell lung cancer

The FDA approved Genentech’s Alecensa Thursday as the first adjuvant treatment for patients with early-stage, anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer who have undergone tumor resection.

In a Phase III study comparing adjuvant Alecensa with platinum-based chemotherapy, Genentech’s drug cut the risk of disease recurrence or death by 76%. Patients also saw significant improvement in central nervous system disease–free survival, according to an exploratory analysis. The study enrolled 257 patients with stage IB to IIIA tumors who had undergone resection and tested positive for ALK.

In a statement Thursday, Genentech CMO Levi Garraway called the disease-free survival data “unprecedented.”

April 18

Product: Takeda’s Entyvio

Indication: Crohn’s disease

The FDA on Thursday approved a subcutaneous (SC) form of Takeda’s Entyvio for maintenance therapy in adults with moderately to severely active Crohn’s disease (CD) after induction therapy with intravenous Entyvio.

The approval was supported by the Phase III VISIBLE 2 study, where a “statistically significant proportion of patients” receiving 108 mg of Entyvio SC maintenance therapy administered every two weeks achieved long-term clinical remission compared to the placebo group after 52 weeks; 48% of people on Entyvio met this primary endpoint vs. 34% of placebo comparators.

Entyvio SC was approved by the FDA in September 2023 for the maintenance treatment of adults with moderately to severely active ulcerative colitis.

April 11

Product: AstraZeneca’s Fasnera

Indication: Asthma

AstraZeneca won a label expansion Thursday for Fasnera, a subcutaneous IL-5 receptor blocker, for the add-on maintenance treatment of severe eosinophilic asthma in kids 6 to 11 years of age.

A severe form of the condition, eosinophilic asthma is caused by inflammation in the respiratory system due to white blood cells called eosinophils. Fasnera binds to the alpha subunit of the human IL-5 receptor, which is typically expressed on the surface of eosinophils and basophils, triggering cell death in both cells, while suppressing inflammation, according to the company.

Fasnera was initially approved in November 2017 for children 12 and older with severe eosinophilic asthma. In a Phase III trial in patients 6 to 12 years, the drug’s pharmacokinetic and pharmacodynamic profiles were consistent with that seen in previous studies, according to AstraZeneca. Fasnera was also found to be safe in the younger population.

April 8

Product: Supernus Pharmaceuticals’ SPN-830

Indication: Parkinson’s disease

Supernus Pharmaceuticals failed, for the second time, to secure approval for its drug-device combo SPN-830, which is intended for the continuous treatment of motor fluctuations in Parkinson’s disease.

In its Complete Response Letter (CRL), the FDA cited undisclosed product quality and master filing issues for the infusion device, which is proprietary to the device manufacturer, according to Supernus’ announcement. No clinical safety or efficacy issues were identified.

This is the second time the FDA has rejected SPN-830. In October 2022, the regulator issued a CRL, requesting additional information and analysis related to the infusion device and drug product “across several areas” of the New Drug Application (NDA).

Jack Khattar, president & CEO of Supernus, said the company “remain[s] committed to bringing SPN-830 to the market” and will work with the FDA to address the issues identified in the CRL and resubmit the NDA.

April 8

Product: ViiV Healthcare’s Dovato

Indication: HIV

ViiV Healthcare picked up another FDA approval for its daily HIV tablet. Dovato, which combines the integrase strand transfer inhibitor dolutegravir with the nucleoside reverse transcriptase inhibitor lamivudine, was approved to treat HIV-1 infection in adolescents 12 years and older, making it the first oral, two-drug, single-tablet regimen available for this patient population, according to ViiV.

ViiV backed the supplemental application with data from the DANCE study, which evaluated Dovato in treatment-naive adolescents, along with evidence from “well-controlled” studies in adults. In DANCE, 26 of 30 participants achieved and maintained viral suppression at week 48, the company reported.

Lynn Baxter, head of North America at ViiV Healthcare, called Dovato’s composition an “important consideration for young people who will require lifelong treatment.”

April 5

Product: AstraZeneca and Daiichi Sankyo’s Enhertu

Indication: HER2-positve solid tumors

AstraZeneca and Daiichi Sankyo notched another victory for Enhertu on Friday, as the antibody-drug conjugate (ADC) became the first approved tumor-agnostic HER2-directed therapy and the first ADC to win such an indication, according to its makers.

The approval was backed by data from the Phase II DESTINY-PanTumor02 trial, which enrolled nearly 470 patients with HER2-positve solid tumors. After a median of 12.75 months, treatment with Enhertu led to an overall response rate of 37.1%, while duration of response was 11.3 months. Patients survived for an average of 6.9 months without disease progression, and 13.4 months overall. Trial participants had a variety of cancers, including biliary tract, bladder, cervical, pancreatic and certain rare tumors.

April 5

Product: Johnson & Johnson and Legend Biotech’s Carvykti

Indication: Multiple myeloma

Johnson & Johnson and Legend Biotech’s Carvykti is now approved for the second-line treatment of multiple myeloma. The FDA’s approval, which came late Friday evening, makes Carvykti the first BCMA-targeted therapy in this treatment line, according to Legend Biotech.

The approval was backed by data from the CARTITUDE-4 study, where treatment with Carvykti led to “statistically significant and clinically meaningful improvement of progression-free survival compared to two standard of care treatment regimens,” according to the companies.

Carvykti carries a boxed warning for potential side effects, including cytokine release syndrome, immune effector cell–associated neurotoxicity syndrome and secondary malignancies.

The FDA’s decision came three weeks after an advisory committee unanimously recommended Carvykti for this patient population. It also follows the approval of Bristol Myers Squibb and 2seventy Bio’s Abecma in third line multiple myeloma.

April 5

Product: Bristol Myers Squibb and 2seventy Bio’s Abecma

Indication: Multiple myeloma

Early Friday, the FDA handed Bristol Myers Squibb and 2seventy Bio a long-awaited approval for their CAR-T therapy Abecma in third line multiple myeloma. Abecma reprograms a patient’s T cells to eliminate cells that express B cell maturation antigen (BCMA), which is present in normal B cells but overexpressed in multiple myeloma. Prior to Friday’s approval, Abecma was authorized to treat patients with relapsed or refractory disease in adult patients who have previously received four or more treatment regimens.

The approval was based on data from the Phase III KarMMa-3 trial, where treatment with Abecma tripled the progression-free survival rate. Patients in the treatment arm survived a median 13.3 months without disease progression, while those in the control group lived only 4.4 months without their disease advancing.

Abecma carries a boxed warning for cytokine release syndrome, neurologic toxicities, HLH/MAS, prolonged cytopenia and secondary hematologic malignancies.

April 4

Product: Basilea Pharmaceutica’ Zevtera

Indication: Staphylococcus aureus bloodstream infections and community-acquired bacterial pneumonia

Fifteen years of persistence paid off for Basilea Pharmaceutica on Thursday, as the FDA approved its antibiotic Zevtera for adults with Staphylococcus aureus bloodstream infections and adult and pediatric patients three months to less than 18 years old with community-acquired bacterial pneumonia.

Data from three studies showed Zevtera has similar efficacy to comparator drugs in the three adult populations. The approval in pneumonia was supported by data from the adult CABP trial and a study in 138 pediatric subjects.

Basilea originally partnered with Johnson & Johnson to develop the molecule in 2005 but ran into a series of regulatory challenges. The Swiss company is seeking a partner to commercialize Zevtera.

April 2

Product: Vanda Pharmaceuticals’ Fanapt

Indication: Bipolar I disorder

Vanda Pharmaceuticals’ Fanapt, originally approved in 2009 for schizophrenia, is now authorized to treat manic or mixed episodes in adults with bipolar I disorder.

Tuesday’s approval is backed by data from a Phase III trial comprising around 400 patients with a history of bipolar I disorder who are currently suffering from a manic episode. Treatment with Fanapt led to significantly greater improvements in the Young Mania Rating Scale, a tool to assess the severity of mania, versus placebo, Vanda reported. Significant superiority over placebo was evident as early as two weeks after treatment initiation. Vanda CEO Mihael Polymeropoulos called the drug a “familiar therapeutic agent that offers flexible dosing with a well-known safety profile.”

April 1

Product: AstraZeneca’s Voydeya

Indication: Paroxysmal nocturnal haemoglobinuria

AstraZeneca kicked off April with a win for its rare disease portfolio as the FDA approved Voydeya, an oral, first-in-class factor D inhibitor to treat a subset of patients with paroxysmal nocturnal haemoglobinuria (PNH). The drug is intended as an add-on therapy for AstraZeneca’s C5 inhibitors Ultomiris and Soliris.

Between 10% and 20% of PNH patients experience clinically significant loss of red blood cells outside of the blood vessels, a condition known as extravascular haemolysis, and continued anemia symptoms when on C5 inhibitors. Voydeya is designed to reduce the need for transfusions and improve outcomes for these patients. In a Phase III trial, the factor D inhibitor led to statistically significant improvement in hemoglobin levels after 12 weeks, improvements that persisted through 48 weeks.

March 28

Product: Akebia’s Vafseo

Indication: Anemia due to chronic kidney disease

After a lengthy regulatory battle, the FDA approved Akebia Therapeutics’ oral treatment vadadustat. The drug, which will be sold under the name Vafseo, will treat adults with anemia due to chronic kidney disease who have been on dialysis for at least three months. While Vafseo is now approved in 37 countries, the FDA did give the oral prolyl hydroxylase inhibitor a boxed warning over a heightened risk of death, stroke, myocardial infection, venous thromboembolism and thrombosis of vascular access.

A publication in the New England Journal of Medicine in 2021 demonstrated that Vafseo’s safety was not significantly worse than darbepoetin alfa regarding the change in mean hemoglobin levels and that it had a similar safety profile as darbepoetin alfa. The FDA also looked at postmarket safety data from Japan, where the drug was launched in 2020.

March 29

Product: Gilead’s Vemlidy

Indication: Chronic hepatitis B in pediatric patients

Gilead has scored a label extension for its antiviral Vemlidy, which is now available for pediatric patients ages six and older with chronic hepatitis B virus (HBV) with compensated liver disease who weigh at least 25 kg. However, the FDA mandated a boxed warning stating that the discontinuation of the therapy may result in severe acute exacerbations of hepatitis B and that hepatic function should be monitored closely. Vemlidy secured approval in 2016 in adults with chronic HBV infection with compensated liver disease and, in 2022, was expanded for treating chronic HBV infection in patients 12 and older.

The FDA expanded Vemlidy due to data from a Phase II trial comparing the drug to a placebo. According to Gilead, patients in the Vemlidy cohort and those on the placebo who switched to open-label Vemlidy after 24 weeks showed “progressive increases” in the rates of virological suppression through 96 weeks.

March 26

Product: Merck’s Winrevair

Indication: Pulmonary arterial hypertension

People with pulmonary arterial hypertension (PAH) now have a therapy that targets the root of their disease after the FDA approved Merck’s Winrevair on Tuesday. Winrevair is the first FDA-approved activin signaling inhibitor therapy for PAH, a mechanism that improves the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation, an underlying cause of the disease, according to Merck’s announcement.

In a Phase III trial, Winrevair hit the primary endpoint, eliciting a statistically significant and clinically meaningful improvement in the 6-minute walk distance test after 24 weeks. Merck expects Winrevair to be available at select pharmacies at the end of April.

March 25

Product: AstraZeneca’s Ultomiris

Indication: Neuromyelitis optica spectrum disorder

Another rare disease, neuromyelitis optica spectrum disorder (NMOSD), saw a new approval Monday as the FDA greenlit AstraZeneca’s Ultomiris for patients positive for anti-AQP4 antibodies. Marc Dunoyer, the company’s rare disease head, in a statement called Ultomiris a “transformative” treatment option for NMOSD, with the “potential to eliminate relapses with a convenient dosing schedule every eight weeks.”

The approval was backed by data from a Phase III trial that met its primary efficacy endpoint, preventing relapse in all 58 treated patients over a median of 73.5 weeks, compared to 20 relapses in the placebo arm of a trial for AstraZeneca’s other NMOSD therapy Soliris. (Given the potential long-term consequences of NMOSD relapses, the company could not ethically include a direct placebo arm in its Phase III trial.) Ultomiris was well-tolerated overall, and most treatment-emergent events were mild or moderate in severity, according to AstraZeneca.

March 25

Product: Regeneron’s odronextamab

Indication: Follicular lymphoma and Diffuse large B-cell lymphoma

Regeneron’s bid for approval of odronextamab for the third-line treatment of relapsed/refractory (R/R) follicular lymphoma and in R/R diffuse large B cell lymphoma, has come up short. On Monday, the company announced that the FDA issued Complete Response Letters (CRL) for both Biologics License Applications.

“The only approvability issue is related to the enrollment status of the confirmatory trials,” according to Regeneron’s announcement. As part of the company’s OLYMPIA program, the FDA is requiring that the trials include both dose-finding and confirmatory portions. While enrollment in the dose-finding portion had begun, “the CRLs indicate that the confirmatory portions of these trials should be underway,” Regeneron stated. The FDA did not cite any issues with clinical efficacy or safety, trial design, labeling or manufacturing.

March 22

Product: J&J’s Opsynvi

Indication: Pulmonary arterial hypertension

Setting up a big week for the pulmonary arterial hypertension (PAH) space, the FDA approved Johnson & Johnson’s single-tablet combination therapy of macitentan and tadalafil for the cardiac disease. Opsynvi is the first once daily combination treatment for PAH, according to J&J. It is intended for patients who are treatment-naive or who have already been exposed to prior endothelin receptor antagonist and phosphodiesterase 5 inhibitors.

In a Phase III study of 187 PAH patients comparing Opsynvi to macitentan and tadalafil monotherapy, J&J’s drug was superior in n reducing pulmonary vascular resistance.

On March 26, the FDA is set to decide on Merck’s sotatercept, which would be the first disease-modifying treatment for PAH.

March 22

Product: AbbVie’s Elahere

Indication: Certain ovarian, fallopian tube and primary peritoneal cancers

Friday, the FDA granted AbbVie’s Elahere full approval for patients with certain ovarian, fallopian tube and primary peritoneal cancers. The nod, which converts a November 2022 accelerated approval, makes Elahere “the first and only antibody-drug conjugate (ADC) approved in the U.S.” for ovarian cancer, Roopal Thakkar, AbbVie’s CMO of global therapeutics, said in a statement.

In a confirmatory Phase III trial comparing Elahere to investigator’s choice of chemotherapy, AbbVie’s drug cut the risk of death by 33% and reduced the likelihood of cancer progression by 35%, the company reported. The study focused on patients with platinum-resistant ovarian cancer whose tumors had high levels of folate receptor alpha.

March 22

Product: Invivyd’s Pemgarda

Indication: COVID-19

There’s another preventative COVID-19 treatment on the market after the FDA greenlit Invivyd’s monoclonal antibody Pemgarda for moderately-to-severely immunocompromised people 12 years and older. Pemgarda is not to be used as a substitute for vaccination, according to Invivyd’s announcement.

Interim data from the ongoing Phase III CANOPY trial showed that Pemgarda could elicit protective titers similar to those produced in the Phase II/III EVADE trial of adintrevimab, its parent antibody. Pre-exposure treatment with Adintrevimab led to a neutralizing titer concentration 90 days after administration, which equated to a 70% clinical efficacy rate.

Pemgarda is expected to be available for order “imminently,” Invivyd CEO Dave Herring in a statement.

March 22

Product: Italfarmaco/ITF’s Duvyzat

Indication: Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) patients have their third new treatment option in nine months after the FDA approved Italfarmaco’s Duvyzat, a nonsteroidal oral drug to be sold by newly incorporated U.S. subsidiary ITF Therapeutics. Duvyzat (givinostat) is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD, according to the FDA’s announcement.

Duvyzat won approval on the strength of a Phase III study that met the primary endpoint of a change in the four-stair climb assessment from the baseline to 72 weeks and showed “favorable outcomes” in key secondary measures, according to Italfarmaco. Craig McDonald, chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health and lead U.S. study investigator, told BioSpace he has treated “a number of patients” with Duvyzat who are still ambulatory at 16 or 17 years old, while DMD patients on steroid treatment typically lose the ability to walk around 10 to 13 years of age.

Duvyzat’s approval follows that of Sarepta’s Elevidys, which got the FDA’s green light in June 2023, and Santhera Pharmaceuticals’ Agamree, which was approved in October.

March 20

Product: Idorsia’s Tryvio

Indication: Hypertension

Wednesday, the FDA greenlit Idorsia Pharmaceuticals’ Tryvio for the treatment of hypertension to reduce blood pressure in adults who are not seeing adequate control on other drugs. Tryvio, which targets the endothelin pathway for hypertension, is intended to be taken alongside other antihypertensive drugs. It is the first FDA-approved medicine in its class for hypertension, according to Idorsia’s announcement.

The approval was supported by data from the Phase III PRECISION trial, where both the 12.5-mg and 25-mg dose levels of Tryvio significantly reduced sitting systolic blood pressure compared with placebo after four weeks of treatment. Only the 12.5-mg dose is approved. The FDA did attach a boxed warning for embryo-fetal toxicity, flagging the risk of major birth defects when used during pregnancy.

Idorsia stated it plans to have Tryvio on the market during the second half of this year.

March 19

Product: Takeda’s Iclusig

Indication: Philadelphia chromosome-positive acute lymphoblastic leukemia

Takeda scored a frontline approval for its kinase inhibitor Iclusig on Tuesday. Iclusig is the first targeted treatment in the U.S. for the frontline treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy, according to Takeda’s announcement.

The supplemental approval is based on data from the Phase III PhALLCON trial, in which treatment with Iclusig led to superior MRD-negative complete remission rates and had comparable safety to Novartis’ Gleevec in adults with newly diagnosed Ph+ ALL. All patients also received reduced-intensity chemotherapy. Iclusig’s label includes a boxed warning for arterial occlusive events, venous thromboembolic events, heart failure and hepatotoxicity.

March 18

Product: Orchard Therapeutics’ Lenmeldy

Indication: Metachromatic leukodystrophy

Monday, the FDA gave a glimmer of hope to families of children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system, with the approval of Orchard Therapeutics’ Lenmeldy.

Lenmeldy is the first FDA-approved gene therapy for MLD, which is caused by a deficiency of the arylsulfatase A (ARSA) enzyme, a state that leads to a buildup of fatty substances in the cells. The disease, which, according to the FDA, affects one in every 40,000 people in the U.S., is characterized by loss of motor and cognitive function and early death. A one-time gene therapy, Lenmeldy is an individualized, single-dose infusion made from a patient’s own hematopoietic stem cells, which have been genetically modified to include functional copies of the ARSA gene.

In two single-arm, open-label clinical trials comprising 37 children with MLD, Lenmeldy “significantly reduced the risk of severe motor impairment or death compared with untreated children,” according to the FDA’s press release.

Orchard Therapeutics was acquired by Kyowa Kirin in October 2023.

March 14

Product: Madrigal’s Rezdiffra

Indication: MASH

In one of the year’s most highly anticipated FDA decisions, Madrigal Pharmaceuticals won approval Thursday for resmetirom—henceforth to be known as Rezdiffra—as the first-ever treatment for metabolic dysfunction-associated steatohepatitis (MASH), formerly noncirrhotic non-alcoholic steatohepatitis (NASH).

The approval, for patients with MASH with moderate to advanced liver scarring, was based on results of the Phase III MAESTRO-NASH trial, where the MASH resolved in 25.9% of patients who received an 80-mg dose of Rezdiffra and 29.9% of those taking 100 mg, with no worsening of fibrosis.

Experts who spoke with BioSpace prior to the approval are hopeful that this small molecule will be a game-changer in terms of both diagnosis of and treatment for MASH.

March 14

Product: BMS’s Breyanzi

Indications: chronic lymphocytic leukemia and small lymphocytic leukemia

As Bristol Myers Squibb made final preparations for Friday’s advisory committee meeting on its CAR-T therapy Abecma, the company got an early win in the same drug class. Late Thursday evening, the FDA approved BMS’s Breyanzi for the treatment of chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL).

Breyanzi won approval in these new indications based on results from the pivotal TRANSCENT CLL 004 trial, where treatment with the CAR-T therapy elicited a complete response rate of 20% and an overall response rate of 45%, with responses lasting more than 35 months. Breyanzi was tagged with a boxed warning for cytokine release syndrome, neurologic toxicities and secondary hematological malignancies.

In an interview with BioSpace, Rosanna Ricafort, vice president and head of late development hematology and cell therapy, said the field has come a long way in terms of toxicity management guidelines “and an understanding of how to manage CAR T-associated toxicities that are well characterized.”

March 14

Product: BeiGene’s Tevimbra

Indication: Esophageal squamous cell carcinoma

Also coming through on a jam-packed Thursday for the FDA was a nod for BeiGene’s PD-1 blocker tislelizumab—which will carry the brand name Tevimbra—in unresectable or metastatic esophageal squamous cell carcinoma. The approval represents Tevimbra’s first in the U.S.; in BeiGene’s native China, it is approved for 11 indications, making it the top PD-1 inhibitor in that country.

Thursday’s approval was supported by data from the RATIONALE 302 study, where Tevimbra led to a statistically significant improvement in overall survival versus the investigator’s choice of chemotherapy. The immunotherapy was also found to have a superior safety profile to chemotherapy, according to BeiGene.

Tevimbra is expected to be available in the U.S. in the second half of this year.

March 13

Product: Mirum’s Livmarli

Indication: Cholestatic pruritus in PFIC

California-based Mirum Pharmaceuticals secured the FDA’s approval for Livmarli for the oral treatment of cholestatic pruritus in patients five years and older with progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder that causes progressive liver disease.

In the Phase III MARCH trial, which enrolled 93 patients across several different genetic subtypes of PFIC, Livmarli significantly improved pruritus symptoms in treated patients, as well as concentrations of serum bile acids and bilirubin.

Mirum CEO Chris Peetz said in a statement that Livmarli could have a “transformational impact for patients with cholestatic pruritis associated with PFIC.”

March 11:

In a press release Monday, Viatris and Mapi Pharma announced that the FDA has rejected their multiple sclerosis hopeful GA Depot 40, which the companies had proposed as a treatment for relapsing forms of the disease.

The development partners are currently reviewing the Complete Response Letter to better determine the “appropriate next steps” for GA Depot and continue to believe in its potential as “an important new treatment advancement for patients with multiple sclerosis,” according to the announcement.

Viatris and Mapi had backed their NDA with data from a Phase III of more than 1,000 patients who received a total of 13 doses of either 40-mg GA Depot or placebo. GA Depot cut the annualized MS relapse rate by 30.1% versus placebo, the companies reported.

March 8:

As if it was possible for Novo Nordisk’s Wegovy to get any hotter, the FDA approved the GLP-1 weight loss drug to reduce the risk of cardiovascular death, heart attack and stroke in adults with cardiovascular disease who are obese or overweight.

In its announcement, which came Friday afternoon, the FDA noted that safety and efficacy of Wegovy in the new indication were based on a trial comprising 17,600 patients randomized to receive either Novo’s drug or placebo, as well as standard medical treatment and lifestyle counseling. In the trial, 6.5% of participants taking Wegovy had a major adverse cardiovascular event compared to 8% on placebo, according to the FDA.

“Wegovy is the first weight-loss drug to also be approved to help prevent life-threatening cardiovascular events in adults with cardiovascular disease and either obesity or overweight,” John Sharretts, director of the Division of Diabetes, Lipid Disorders and Obesity in the FDA’s Center for Drug Evaluation and Research, said in a statement.

March 7:

BeiGene won its fifth FDA approval for Brukinsa on Thursday, making it the first Bruton’s tyrosine kinase (BTK) inhibitor to secure five oncology indications, according to BeiGene’s announcement. Brukinsa, in combination with Genentech’s Gazyva, is now authorized for the third-line treatment of relapsed or refractory follicular lymphoma (FL). Brukinsa is also the first BTK inhibitor approved to treat FL.

The FDA’s decision was supported by data from the Phase II ROSEWOOD trial, in which the Brukinsa–Gazyva combo led to a 69% overall response rate (ORR) compared to 46% ORR in patients treated with Gazyva alone, according to BeiGene. The safety profile in the trial was consistent with what had been previously established for both treatments.

March 7:

Chalk up another indication for Opdivo as well, as Bristol Myers Squibb announced the approval Thursday of the blockbuster immunotherapy, in combination with cisplatin and gemcitabine, for unresectable or metastatic bladder cancer. The Opdivo combo is the first concurrent immunotherapy-chemotherapy combination approved for this patient population in the U.S., according to BMS’s press release.

The FDA’s approval was based on results from the Phase III CheckMate–901 trial, where the combo regimen demonstrated a statistically significant improvement in overall survival and progression-free survival compared to cisplatin-gemcitabine alone, BMS reported. Patients treated with Opdivo plus the two chemo agents and followed for approximately 33 months saw a 22% reduction in risk of death compared with chemotherapy alone. Risk of disease progression or death was cut by 28%.

March 5:

Amgen’s bone-preserving therapy denosumab picked up two new competitors on Tuesday as the FDA approved Sandoz’s biosimilars Jubbonti and Wyost. Jubbonti will go up against Amgen’s Prolia for the treatment of osteoporotic men and postmenopausal women who are at high risk of fracture, while Wyost will challenge Amgen’s Xgeva in the prevention of skeletal-related events in patients with multiple myeloma and solid tumors with bone metastases, among other indications.

The two biosimilars also hold the FDA’s interchangeability designation, which allows them to be substituted for their reference products without a change in the patient’s prescription. Sandoz has not disclosed a launch timeline or pricing for either drug.

March 4:

Eyenovia and development partner Formosa Pharmaceuticals secured entry into a $1.3 billion annual market with the FDA approval of their clobetasol propionate 0.05% eye drops for post-operative inflammation and pain after eye surgery.

The product is the first ophthalmic clobetasol propionate drug to be approved by the FDA, and the first steroid to enter the ophthalmic space in more than 15 years, according to the companies’ announcement. The approval was supported by two pivotal Phase III trials that showed the clobetasol propionate eye drops could rapidly and significantly clear ocular inflammation and relieve ocular pain within 14 days of treatment. The product was well-tolerated and had a safety profile similar to placebo.

March 4:

Turn that frown upside down. The FDA on Monday approved a new treatment for frown lines that appear in between the eyebrows in Hugel America’s neurotoxin injection letibotulinumtoxinA-wlbg, which will be marketed as Letybo.

Letybo’s approval is supported by data from three Phase III trials comprising more than 1,200 participants and showing that the drug elicited significant treatment success, defined as no or mild glabellar lines and an improvement of at least two points on the Glabellar Line Scale versus baseline, according to its label. Letybo carries a boxed warning for the distant spread of the toxin’s effect to other body parts. “Swallowing and breathing difficulties can be life threatening and there have been reports of death,” the label reads.

March 1:

The FDA on Friday granted full approval of J&J’s Rybrevant in combination with chemotherapy as a first-line treatment for patients with non-small cell lung cancer (NSCLC) EGFR exon 20 insertion mutations, the company announced. Rybrevant is the “first targeted approach approved for the first-line treatment” in this patient population, Kiran Patel, vice president of clinical development, solid tumors at J&J Innovative Medicines, said in a statement.

The approval is based on data from the randomized, open-label Phase III PAPILLON trial that showed the Rybrevant-based regimen cut the risk of disease progression or death by 60% versus chemotherapy alone. Treatment with the combo also “significantly improved” objective response rate, according to an October 2023 readout. As of that readout, overall survival showed a positive trend in favor of the Rybrevant combo, though this result did not reach statistical significance.

Rybrevant won accelerated approval in May 2021 for the second-line treatment of this patient population.

Feb 27:

In one of the more closely watched decisions of the first quarter of 2024, the FDA rejected Minerva Neurosciences’ roluperidone, which had been proposed to treat the negative symptoms of schizophrenia. While one study did show statistical significance, the FDA said in a Complete Response Letter that it was “insufficient on its own [to provide] substantial evidence of effectiveness.”

The FDA also noted that the NDA submission lacked data on concomitant antipsychotic administration, did not include data that established that the change in negative symptoms was clinically meaningful and that the submitted safety database had an “inadequate number of subjects exposed to roluperidone” at the proposed dose of 64 mg for at least 12 months.

Minerva will “request a meeting to discuss the issues raised and attempt to address FDA’s feedback,” CEO Remy Luthringer said in a statement.

Feb. 27:

A week after the FDA denied Venatorx Pharmaceuticals and Melinta Therapeutics’ New Drug Application for urinary tract infection (UTI) antibiotic cefepime-taniborbactam, competitor Allecra Therapeutics won approval of Exblifep in the same indication. The approval was granted based on data that showed the drug’s effectiveness against antimicrobial resistance in gram-negative bacteria, especially resistance mediated by both extended spectrum beta lactamases and AmpC, according to Allecra’s announcement.

Results from a Phase III trial met the criteria for non-inferiority and superiority compared to piperacillin/tazobactam in the primary outcome of clinical cure and microbiological eradication in patients with complicated UTIs.

Along with the approval, the German company receives a five-year marketing exclusivity extension from the FDA as part of the Generating Antibiotic Incentives Now (GAIN) Act.

Feb 24:

This past weekend brought bad news for Oncopeptides as the FDA announced it would withdraw the approval of Pepaxto, which was greenlit in March 2021, in combination with dexamethasone, to treat certain patients with multiple myeloma.

The FDA made its decision to withdraw approval after a new study failed to confirm Pepaxto’s clinical benefit. The available evidence demonstrated that the drug “is not shown to be safe or effective under its conditions of use,” according to regulator’s announcement. The decision is effective immediately.

Feb. 23:

AbbVie’s Humira got hit with another biosimilar on Friday as the FDA approved Alvotech and Teva’s Simlandi (adalimumab-ryvk). Notably, Simlandi is the first high-concentration, citrate-free and interchangeable Humira biosimilar to be approved in the U.S., according to Alvotech’s announcement. Like Humira, Simlandi is approved for a laundry list of conditions: rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, Crohn’s disease, ulcerative colitis, ankylosing spondylitis, plaque psoriasis, uveitis and hidradenitis suppurativa.

For Alvotech and Teva, the victory was hard-won. In April 2023, the FDA issued a Complete Response Letter for the biosimilar, citing manufacturing issues at its Reykjavik, Iceland, facility. Another CRL came two months later, when the FDA rejected the companies’ bid for the interchangeability designation.

Feb. 23:

CMC issues tripped up Venatorx Pharmaceuticals and Melinta Therapeutics in their quest to gain FDA approval for the urinary tract infection antibiotic cefepime-taniborbactam. On Friday, the development partners said in a press release announcing the Complete Response Letter that the FDA requested additional CMC and related data about the drug, testing methods and manufacturing process. The regulator did not cite any issues related to the clinical safety or efficacy provided in the New Drug Application for cefepime-taniborbactam, a beta-lactam/beta-lactamase inhibitor combination antibiotic intended to treat adults with complicated urinary tract infections.

The companies are “hard at work generating the additional requested CMC data,” Venatorx CEO Christopher Burns said in a prepared statement.

Feb. 16:

Capping a busy Friday afternoon, the FDA approved AstraZeneca’s Tagrisso in combination with chemotherapy to treat advanced non-small cell lung cancer (NSCLC) patients harboring EGFR mutations. The approval marks the fourth NSCLC indication for Tagrisso, which is also authorized for the first-line treatment of metastatic disease, as an adjuvant therapy after resection and in metastatic NSCLC that has progressed after EGFR tyrosine kinase inhibitor (TKI) treatment.

The new approval is based on data from the Phase III FLAURA 2 study, where Tagrisso plus chemotherapy prolonged progression-free survival by around nine months compared to Tagrisso alone.

Dave Fredrickson, executive vice president of AstraZeneca’s oncology business unit, said in a statement that the approval reinforces Tagrisso “as the backbone of EGFR-mutated lung cancer treatment either as a monotherapy or in combination with chemotherapy.”

Feb. 16:

Friday afternoon, the FDA announced one of 2024’s most-anticipated approvals: that of Iovance Biotherapeutics’ lifileucel for advanced melanoma. Lifileucel—which will be known commercially as Amtagvi—is the first one-time cell therapy for a solid tumor and the first tumor-infiltrating lymphocytes (TIL) therapy to be approved by the FDA.

Of 73 patients treated with Amtagvi, 31.5% saw an objective response, with three experiencing a complete response and 20 a partial response. The TIL therapy carries a boxed warning for treatment-related mortality, prolonged severe cytopenia, severe infection, and cardiopulmonary and renal impairment.

“The approval of this first in a new class of cell therapies for solid tumors defines a regulatory path for TIL, which will unlock more innovation in the industry and ultimately benefit patients with cancer,” Jason Bock, CEO of CTMC, a joint venture between biotech Resilience and MD Anderson Cancer Center, told BioSpace in an email.

Feb. 16:

Novartis and Roche’s Xolair became the first treatment for children and adults with one or more food allergies on Friday as the FDA approved the blockbuster to treat accidental exposure in patients one year and older with IgE-mediated food allergies.

The regulatory green light was based on results from the Phase III OUtMATCH study, which assessed Xolair in patients with allergies to peanuts and two other food allergies, including milk, egg, wheat, cashew, hazelnut and walnut. At the beginning of the study, patients were unable to tolerate up to 100 mg of peanut protein. After 16 to 20 weeks of treatment with Xolair, 68% of patients could handle at least 600 mg of peanut protein without moderate to severe allergic symptoms. This was compared to 5% of patients who received a placebo, a difference that was deemed statistically significant.

Feb 15:

EMD Serono, a Merck KGaA company, got a win Thursday as the FDA converted Tepmetko’s February 2021 accelerated nod to full approval for adult non-small cell lung cancer (NSCLC) patients whose disease harbors mesenchymal-epithelial transition (MET) exon 14 skipping alterations.

The accelerated approval was granted based on initial overall response rate (ORR) of 57% in 164 treatment-naïve patients and duration of response (DOR)—40% of responders had a duration of response greater than 12 months, according to the FDA’s full approval announcement. The conversion to traditional approval is based on an additional 161 patients and another 28 months of follow-up time to assess DOR.

Feb. 14:

With much of the American Northeast digging out from an epic snowstorm, the FDA approved the first-ever treatment for severe frostbite in Eicos Sciences’ Aurlumyn (vasodilator iloprost). Iloprost is a vasodilator whose mode of action is to loosen up blood vessels and prevent clots from forming.

“Having this new option provides physicians with a tool that will help prevent the life-changing amputation of one’s frostbitten fingers or toes,” said Norman Stockbridge, director of the Division of Cardiology and Nephrology at the FDA’s Center for Drugs and Evaluation Research, in a statement.

An open-label study enrolled 47 severely frostbitten adults treated with intravenous aspirin plus standard of care. Patients were randomly assigned to receive Aurlumyn intravenously for six hours a day for up to eight days or unapproved frostbite interventions with or without Aurlumyn. Patients treated with Aurlumyn alone saw a 0% risk of amputation compared with 60% of those who did not receive the new treatment.

Feb. 13:

Patients with a particularly deadly cancer got their first new frontline treatment in more than a decade as the FDA approved Ipsen’s Onivyde, along with three chemo drugs, for metastatic pancreatic adenocarcinoma (mPDAC). The The Onivyde-based regimen, dubbed Nalirifox, is intended for newly diagnosed patients with mPDAC.

The approval is based on the Phase III NAPOLI 3 trial, where the Nalirifox regimen—which combines Onivyde with fluorouracil, oxaliplatin and leucovorin—boosted overall survival (OS) by a statistically significant 17% compared to standard of care. Nalirifox also improved progression-free survival by 31%. According to Ipsen, NAPOLI 3 is the first positive Phase III trial in mPDAC to show better OS compared to the current standard-of-care regimen, which consists of nab-paclitaxel and gemcitabine.

Onivyde, in combination with fluorouracil and leucovorin, was first approved in 2015 for patients who had progressed after treatment with gemcitabine.

Feb. 12:

Takeda is on a mini winning streak. After scoring another indication for its immunoglobulin therapy, GAMMAGARD LIQUID, at the end of January, the Japanese pharma on Monday announced the FDA approval of Eohilia for eosinophilic esophagitis (EoE). With the nod, Eohilia becomes the first the first oral treatment for EoE, a chronic allergic condition of the esophagus, according to Takeda’s announcement. Eohilia, a corticosteroid, is intended for 12 weeks of treatment in patients 11 years and older.

The approval is based on a pair of multicenter, placebo-controlled 12-week studies that showed “significantly more patients” taking Eohilia achieved histologic remission vs. placebo, according to the press release. In the first study, 53.1% of patients in the treatment group entered remission vs. 1% of the placebo group; in the second study, the result was 38% vs. 2.4%.

Jan. 29:

Takeda secured another indication for its immunoglobulin therapy, GAMMAGARD LIQUID, which, according to the company, is the only intravenous IG (IVIG) therapy for the treatment of multiple neuromuscular disorder indications in the U.S.

The approval for Chronic Inflammatory Demyelinating Polyneuropathy (CDIP), announced Monday, was backed by data from the ADVANCE-CIDP 2 clinical trial, where treatment with GAMMAGARD LIQUID yielded a 94.4% responder rate, indicating a significant improvement in functional disability.

GAMMAGARD LIQUID is also indicated for multifocal motor neuropathy to boost muscle strength and disability and as a replacement therapy for primary immunodeficiency in patients two years and older.

Jan. 25:

Regeneron and Sanofi’s Dupixent (dupilumab) racked up another indication Thursday, becoming the first and only medicine approved for kids one year and older with eosinophilic esophagitis (EoE), according to Regeneron’s announcement.

Dupixent was approved in May 2022 to treat patients 12 and older with EoE, a progressive disease partly caused by inflammation that damages the esophagus and impairs its function.

The expanded approval was based on data from the Phase III EoE KIDS trial, where 66% of children who received the higher dose Dupixent at tiered dosing regimens experienced histological disease remission after 16 weeks of treatment, compared to just 3% in the placebo group, per the press release. This remission was sustained in 53% of patients in both of the trial’s cohorts after 52 weeks with Dupixent.

Jan. 24:

The FDA has denied Theratechnologies’ supplemental Biologics License Application for an F8 formulation of tesamorelin, which is intended to reduce excess abdominal fat in patients with HIV and lipodystrophy. A number of issues tripped up the Montreal–based biotech, according to its press release announcing the Complete Response Letter, including chemistry, manufacturing and controls issues. The FDA’s questions specifically relate to microbiology, assays, impurities and stability for both the lyophilized product and the final reconstituted drug product. The regulator also requested more information on the potential effects of the more concentrated formulation of tesamorelin on immunogenicity risk.

Theratechnologies will “address these new comments as swiftly as possible,” CMO Christian Marsolais said in a prepared statement.

Tesamorelin first won FDA approval in 2010, and an F4 formulation—which is four times more concentrated than the original drug product—hit the market in 2018 as Egrifta SV. The F8 formulation would allow for smaller administration volumes.

Jan. 19:

Johnson & Johnson’s FGFR kinase inhibitor Balversa (erdafitinib) received full FDA approval Friday for locally advanced or metastatic urothelial carcinoma, converting an accelerated nod granted in April 2019. Specifically, Balversa is approved to treat patients whose cancer has susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations and who have progressed after at least one line of systemic therapy.

At the time of its 2019 approval, Balversa was the first FDA-authorized FGFR inhibitor. It works by targeting and binding to FGFR1, FGFR2, FGFR3 and FGFR4, blocking their enzymatic activity, according to J&J’s website.

The confirmatory Phase III THOR trial pitted Balversa against the standard of care regimen, consisting of chemotherapy or Merck’s Keytruda. In a cohort of patients who had undergone at least one prior line of anti-PD-(L)1 therapy, Balversa cut the risk of death by 36% versus chemotherapy. Patients receiving Balversa also lived a median of four months longer than those on chemo, a result deemed statistically significant.

Jan. 18:

Chemistry, manufacturing and controls issues cost Satsuma Pharmaceuticals as the FDA issued a Complete Response Letter for its nasal powder migraine drug, STS101, according to a Thursday press release by parent company Shin Nippon Biomedical Laboratories. Satsuma will discuss the rejection with the FDA, building toward a resubmission. No safety issues were identified in the CRL, nor did the FDA request any additional studies be conducted, according to Endpoints News.

STS101 is a reformulated version of dihydroergotamine mesylate (DHE), a well-established migraine medication for nasal administration. The product uses Satsuma’s proprietary device for delivery.

Jan. 16:

One of the most-anticipated FDA decisions of the first half of 2024 came early as the regulator approved Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) as a one-time treatment for transfusion-dependent beta-thalassemia (TDT). The green light came more than two months ahead of Casgevy’s March 30 PDUFA date in TDT and just one month after it made history as the first approved CRISPR-based gene-editing therapy, for sickle cell disease.

Vertex is currently working to get the infrastructure up and running for Casgevy, collaborating with hospitals to establish a network of authorized treatment centers (ATCs). More ATCs will be coming online “in the coming weeks,” according to its announcement.

Jan. 16:

Takeda on Tuesday won a label expansion for HyQvia to treat the rare, acquired and immune-mediated disorder chronic inflammatory demyelinating polyneuropathy (CIDP). The FDA has authorized HyQvia (immune globulin infusion 10% with recombinant human hyaluronidase) as a maintenance treatment to prevent the relapse of neuromuscular disability and impairment in adult patients with CIDP.

HyQvia’s label expansion is based on data from the randomized, placebo-controlled ADVANCE-CIDP 1 trial and single-arm open-label extension ADVANCE-CIDP 3 trial, each of which revealed a favorable safety profile for the treatment. In ADVANCE-CIDP 1, HyQvia reduced the rate of relapse by more than 18% versus placebo, an effect that was deemed statistically significant.

HyQvia was first approved in September 2014 for primary immunodeficiency.

Jan. 12:

Merck’s Keytruda won its third approval in cervical cancer and 39th overall Friday when the FDA approved the blockbuster immunotherapy, in combination with chemoradiotherapy, to treat patients with International Federation of Gynecology and Obstetrics (FIGO) 2014 Stage III-IVA disease.

Keytruda is the first anti-PD-1 therapy approved in combination with chemoradiotherapy (CRT) for this indication, according to Merck’s announcement. The FDA’s approval was backed by data from the Phase III KEYNOTE-A18 study, where Keytruda plus CRT reduced the risk of disease progression or death by 41% versus placebo plus CRT, Merck reported. Median progression-free survival was not reached in either group.

In a prepared statement, Bradley Monk, an oncologist and professor of obstetrics and gynecology at the University of Arizona’s College of Medicine and Creighton University School of Medicine, said the approval “has important implications for the way we treat [these patients] moving forward.”

Keytruda is also approved to treat persistent, recurrent or metastatic PD-L1-positive cervical cancer in combination with chemotherapy and as a monotherapy for recurrent or metastatic cervical cancer that has progressed despite chemotherapy.

Jan 8:

Astellas has failed to gain approval for what would be the first approved therapy targeting claudin 18.2 (CLDN18.2), a transmembrane protein expressed on the surface of gastric epithelial cancer cells. “Unresolved deficiencies” at a third party manufacturing facility are what tripped Astellas up in its bid for approval of zolbetuximab, according to the company’s announcement of the Jan. 4 Complete Response Letter.

Zolbetuximab is being proposed to treat patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are CLDN18.2 positive.

Jan 5:

Patients with a highly contagious skin condition called molluscum contagiosum have their first at-home treatment option after the FDA approved Ligand Pharmaceuticals’ Zelsuvmi, a topical nitric oxide-releasing agent. The active ingredient in Zelsuvmi, berdazimer sodium, was developed at Novan, with funding assistance from Ligand, which bought the drug, along with another asset, for $12.2 million when Novan filed for bankruptcy.

Zelsuvmi won approval on the strength of Phase II and III trials where it was well-tolerated and reduced lesion counts when used once a day, according to a press release issued by Ligand. Zelsuvmi, which is approved for individuals one year and older, is expected to be available in the U.S. in the second half of 2024.

Dec 26:

Accelerated approval doesn’t always equal full clearance, something Amgen was reminded of when the FDA declined to convert Lumakras’ May 2021 conditional nod in KRASG12C-mutated non-small cell lung cancer (NSCLC) to a traditional one.

The decision likely didn’t come as a complete surprise to Amgen, as it followed a 10-2 advisory committee vote in October against the drug. The FDA’s Oncologic Drugs Advisory Committee argued that the data from the confirmatory Phase III CodeBreaK 200 study could not reliably be interpreted. Amgen will now be required to run an additional confirmatory study to be completed no later than February 2028, the company stated in a press release announcing the Complete Response Letter.

Dec 23:

The holidays didn’t get off to a happy start for Zealand Pharma, which received a Complete Response Letter for dasiglucagon, which the company intends to treat hypoglycemia in pediatric patients with congenital hyperinsulinism (CHI). The CRL is specifically for part one of the New Drug Application, in which Zealand sought approval for dasiglucagon for the prevention and treatment of hypoglycemia in pediatric patients 7 days of age and older with CHI for up to 3 weeks of dosing.

The CRL is related to deficiencies identified following an inspection at a third-party contract manufacturing facility, Zealand noted in a press release, adding that the deficiencies are not specific to dasiglucagon itself. The company intends to resubmit the NDA during the first half of this year after a successful reinspection of the manufacturing site.

Dec 21:

The FDA on Thursday signed off on the first and only self-administered treatment for polyneuropathy in hereditary transthyretin-mediated amyloidosis (hATTR-PN): AstraZeneca and Ionis’ eplontersen, now to be known as Wainua. The approval was granted based on data from the Phase III NEURO-TTRansform study, where treatment with Wainua significantly reduced serum concentrations of the transthyretin (TTR) protein and significantly improved neuropathy impairment.

Wainua is a ligand-conjugated antisense oligonucleotide therapy that works by tagging the TTR mRNA for degradation, reducing the overall production and concentration of the TTR protein. It can be self-administered with an auto-injector.

Dec 20:

The FDA on Wednesday granted full approval to Calliditas Therapeutics’ Tarpeyo (budesonide) delayed release capsules for adults with primary immunoglobulin A nephropathy (IgAN). Tarpeyo won accelerated approval in December 2021.

In the Phase III NefIgArd trial, patients treated with Tarpeyo saw significantly better kidney function, as measured by the estimated glomerular filtration rate (eGFR) versus placebo after nine months on the drug and 15 months of follow-up. After two years of treatment, patients receiving Tarpeyo saw 50% less deterioration in kidney function than those in the placebo arm.

According to Calliditas’ announcement, Tarpeyo is the only FDA-approved treatment for IgAN to significantly reduce the loss of kidney function.

Dec 20:

Merck just can’t get gefapixant across the regulatory finish line. On Wednesday, the FDA issued a Complete Response Letter, stating that Merck’s data package “did not meet substantial evidence of effectiveness” for the treatment of refractory chronic cough (RCC) or unexplained chronic cough (UCC) in adults.

The rejection—Merck’s second in nearly two years—was not a complete surprise. Last month, the FDA’s Pulmonary-Allergy Drugs Advisory Committee voted 12-1 that the evidence supplied by Merck did not show a clinically meaningful benefit in the intended population.

Merck will review the FDA’s letter and feedback to determine the next steps for gefapixant, according to Wednesday’s announcement of the CRL.

Dec 19:

A rare skin disease affecting young children has a new treatment. On Tuesday, the FDA approved Chiesi Global Rare Diseases’ Filsuvez (triterpenes) to treat wounds related to junctional epidermolysis bullosa (EB).

Filsuvez won approval based on data from the Phase III EASE study, where the drug led to accelerated wound healing, along with a reduction in overall wound burden and less pain associated with changing wound dressings.

Filsuvez, a topical herbal gel mixture containing dry extracts from two species—Betula pendula Roth and Betula pubescens Ehrh and hybrids of the two—was developed by Amryt Pharma, which Chiesi acquired in January of this year for $1.25 billion upfront.

Dec 18:

Inspection issues have, at least temporarily, quashed Checkpoint Therapeutics’ hopes of an approval for cosibelimab in metastatic or locally advanced cutaneous squamous cell carcinoma (cSCC). In a Complete Response Letter issued Monday, the FDA cited findings during a multi-sponsor inspection of Checkpoint’s third-party contract manufacturer that the company will need to address in its resubmission, Checkpoint said in a press release.

Checkpoint is confident, however, that it will prevail next year. “As the only deficiencies relate to the FDA’s inspection of our third-party contract manufacturing organization, we believe we can address the feedback in a resubmission to enable marketing approval in 2024,” James Oliviero, the company’s president and CEO, said in a statement.

Dec 15:

This cancer collaboration just keeps on giving. On Friday, the FDA approved the combination of Merck’s Keytruda and Astellas and Pfizer/Seagen’s Padcev for the first-line treatment of locally advanced or metastatic urothelial cancer.

In a Phase III trial of 886 patients, Keytruda, a PD-1 inhibitor, plus Padcev, an antibody-drug conjugate, reduced the risk of death by 53% over platinum-based chemotherapy. Median overall survival was 31.5 months for the combo versus 16.1 months for chemotherapy. Median progression-free survival for Keytruda plus Padcev was 12.5 months compared to 6.3 months for chemo alone. These results were good enough for a very early approval—the companies’ supplemental Biologics License Application was accepted not three weeks ago on Nov. 30, when the FDA set a PDUFA date of May 9, 2024.

Dec 15:

On Friday, the FDA approved Zoryve (roflumilast) topical foam, developed by Arcutis Biotherapeutics to treat patients 9 years and older with seborrheic dermatitis. According to Arcutis’ press release, Zoryve is the first drug in more than two decades with a new mechanism of action for seborrheic dermatitis, a skin condition affecting primarily the scalp.

In the pivotal STRATUM trial, nearly 80% of individuals achieved the primary efficacy endpoint of Investigator’s Global Assessment (IGA) Success, with just over 50% reaching complete clearance at week 8.

Seborrheic dermatitis, which affects more than 10 million people in the United States, is characterized by red patches covered with large, greasy, flaking yellow-gray scales and persistent itch, according to Arcutis.

Dec 14:

Merck scored a second approval for HIF-2α inhibitor Welireg (belzutifan), this one in advanced renal cell carcinoma (RCC). Specifically, Welireg is approved for RCC patients who have already been treated with a PD-1 or PD-L1 inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor.

The approval is based on the Litespark-005 trial, where Welireg surpassed Novartis’ Afinitor (everolimus), Merck stated in a press release. Treatment with Welireg led to an objective response rate of 22% versus 4% for those treated with Afinitor. Welireg represents the first novel therapeutic class to be approved in advanced RCC since 2015, according to Merck.

Welireg was first approved in 2021 to treat adults with von Hippel-Lindau disease, a genetic condition that causes non-cancerous tumors in the body to grow.

Dec 14:

High-risk neuroblastoma (HRNB) patients now have an oral maintenance therapy option after the FDA greenlit US WorldMeds’ Iwilfin (eflornithine). Iwilfin is intended to reduce the risk of disease relapse in both adult and pediatric patients.

Iwilfin, an orally available inhibitor of ornithine decarboxylase, was approved based on the results of a single-arm study of children with HRNB who were initially treated with standard regimens, including immunotherapy. Results of the study showed an 84% event-free survival rate after four years compared with 73% in an external control arm. Overall survival after treatment with Iwilfin was 96% after four years versus 84% in the external control group.

Iwilfin led to a drop in expression levels of two oncogenic drivers—MYCN and LIN28B—both known to promote cell aging and prevent cancer development, according to the drug’s label.

Dec 8:

In an historic moment Friday, the FDA approved the first-ever gene therapy based on the Nobel Prize-winning CRISPR/Cas9 gene editing system discovered more than a decade ago by Jennifer Doudna and Emmanuelle Charpentier.

Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has been hailed as curative for sickle cell disease (SCD) patients. Data from a pivotal Phase I/II/III trial and long-term safety and efficacy study together showed the therapy could significantly lessen severe vaso-occlusive events and hospitalizations. Vertex and CRISPR have priced Casgevy at $2.2 million.

SCD patients got not one but two new therapies Friday—the first gene therapies for the disorder caused by a mutation in the genes involved in producing hemoglobin beta—when the FDA approved bluebird bio’s Lyfgenia nearly two weeks ahead of its Dec. 20 action date. Lyfgenia (lovo-cel), a lentiviral gene therapy, led to complete resolution of vaso-occlusive events in 28 of 32 patients. Bluebird has set a list price of $3.1 million for Lyfgenia.

Dec 5:

Another rare disease saw an FDA approval Wednesday as the regulator approved Novartis’ Fabhalta (iptacopan) for paroxysmal nocturnal hemoglobinuria (PNH).

Fabhalta inhibits Factor B, which plays a key role in the proximal alternative pathway of the complement cascade. In PNH, heightened activity of the complement system prematurely destroys red blood cells, causing symptoms such as anemia and thrombosis. In a Phase III study, 82.3% of patients—who had residual anemia after anti-C5 treatment—saw a sustained increase in hemoglobin levels even without transfusions compared with 0% of those who stayed on anti-C5 treatments.

As of Dec. 4, 37 rare disease treatments had been approved by the FDA in 2023, according to stats provided to BioSpace by the National Organization for Rare Disorders.

Dec 1:

Eli Lilly can add two more indications for Jaypirca (pirtobrutinib) as the FDA greenlit the reversible Bruton’s tyrosine kinase (BTK) inhibitor for chronic lymphocytic leukemia or small lymphocytic leukemia. Jaypirca is specifically intended for adults who have undergone at least two prior lines of therapy, including another BTK inhibitor and a BCL-2 inhibitor.

Jaypirca was first approved for relapsed or refractory mantle cell lymphoma in January. That approval broke new ground, as Jaypirca was the first-ever reversible BTK inhibitor to be authorized by the FDA. It is still the only such drug on the market.

Nov 27:

SpringWorks Therapeutics and people with desmoid tumors—a rare type of non-cancerous tumor that can be locally aggressive—have a new treatment to be thankful for. On Monday, the FDA approved nirogacestat, to be marketed as Ogsiveo, as the first-ever therapy for this potentially painful and physically debilitating condition.

In a pivotal trial of 142 adult patients with progressing desmoid tumors not amenable to surgery, Ogsiveo elicited “clinically meaningful and statistically significant improvement” in the primary endpoint of progression-free survival compared to placebo. An additional efficacy measure, objective response rate, was also statistically different, with 41% of patients receiving Ogsiveo seeing a response versus just 8% of those on placebo.

Nov 27:

As the company anticipated and warned investors last month in an SEC filing, the FDA on Monday rejected Aldeyra Therapeutics’ dry eye disease candidate. In a Complete Response Letter, the regulator stated that Aldeyra’s New Drug Application did not demonstrate “efficacy in treating ocular symptoms associated with dry eyes” and informed the company that “at least one additional adequate and well-controlled study” would be required to demonstrate efficacy.

Aldeyra has already submitted a Special Protocol Assessment for a crossover clinical trial and expects to hear FDA feedback on the plan in December. Depending on this feedback and assuming that the results of this trial are positive, Aldeyra intends to resubmit the NDA during the first half of 2024.

Nov 17:

AstraZeneca scored a first-in-class approval for its pan-AKT inhibitor capivasertib as a treatment for adults with hormone receptor–positive and HER2-negative locally advanced or metastatic breast cancer whose tumors also have PIK3CA, AKT1 or PTEN mutations. Dubbed Truqap, the new treatment is approved in combination with Astra’s Faslodex.

The FDA’s green light was based on results from the Phase III CAPItello-291 where the combination cut the risk of disease progression or death by half compared to Faslodex alone.

“The rapid U.S. approval of Truqap reinforces the important role of the PI3K/AKT/PTEN pathway in HR-positive breast cancer and the critical need to test patients at the time of diagnosis, as up to fifty percent have tumors with these alterations,” Dave Fredrickson, executive vice president of Astra’s oncology business unit, said in a prepared statement.

Nov 17:

Astellas and Pfizer won FDA approval Friday for Xtandi, an androgen receptor signaling inhibitor, to treat nonmetastatic castration-sensitive prostate cancer (nmCSPC). Xtandi—which is also approved for four other types of prostate cancer—is the first androgen receptor signaling inhibitor authorized to treat nmCSPC with biochemical recurrence at high risk for metastasis, according to the companies’ announcement.

The supplemental approval was granted based on data from the Phase III EMBARK trial, which studied Xtandi plus leuprolide, placebo plus leuprolide (a synthetic hormone regulator) and Xtandi as a monotherapy. After five years, patients treated with the combination had a metastasis-free survival of 87.3%. This was compared to 80% in the Xtandi monotherapy arm and 71.4% in the leuprolide-only cohort.

Nov 16:

Merck’s Keytruda racked up another indication Thursday, this time for the first-line treatment of stomach cancer. Specifically, Keytruda was approved in combination with chemotherapy to treat adults with locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma. The approval marks the seventh for the anti-PD-1 superstar in gastrointestinal cancer and its 38th overall in the U.S.

The first-line nod was given based on data from the Phase III KEYNOTE-859 study, where the Keytruda-based regimen cut the risk of death by 22% versus chemotherapy alone. Median survival was just over a month longer with Keytruda treatment at 12.9 months versus 11.5 months.

Nov 15:

Patients with ROS1-positive non-small cell lung cancer have a new treatment option after the FDA approved Bristol Myers Squibb’s Augtyro (repotrectinib) on Wednesday. A particularly aggressive form of lung cancer, ROS-positive NSCLC is difficult to treat and can often spread to the brain.

In the pivotal TRIDENT-1 study, Augtyro, a tyrosine kinase inhibitor (TKI) targeting ROS1 oncogenic fusions, led to an objective response rate of 79% in TKI-naive patients. Among patients pretreated with one prior ROS1 TKI and no prior chemotherapy, the ORR was 38%. The median duration of response (mDOR) for TKI-naive patients was 34.1 months and 14.8 months for the pretreated group.

Nov 15:

Patients with kidney failure receiving chronic hemodialysis are vulnerable to contracting catheter-related bloodstream infections. On Wednesday, the FDA greenlit a treatment designed to reduce these infections: CorMedix’s DefenCath, a combination of the amino acid derivative taurolidine and the anticoagulant heparin.

In a Phase III trial, DefenCath lowered the incidence of catheter-related bloodstream infections by 71% versus heparin alone. The efficacy results were so impressive that an Independent Data Safety and Monitoring Board recommended the study’s early termination.

It has not been an easy path to the finish line for CorMedix, which was previously turned away twice by the FDA. Both Complete Response Letters were due to manufacturing and supplier issues. CorMedix expects DefenCath to be available in the inpatient setting in the first quarter of 2024.

Nov 9:

Thursday, the FDA approved Valneva’s Ixchiq as the first vaccine to prevent disease caused by the chikungunya virus. Ixchiq is intended for adults at increased risk of exposure to the virus, which is typically transmitted through the bite of an infected mosquito. Those at highest risk live in the tropical and subtropical regions of Africa, Southeast Asia and parts of the Americas, according to the FDA’s announcement.

Safety of Ixchiq was demonstrated in two clinical studies made up of approximately 3,500 participants; in one study, around 1,000 people received a placebo. The most common side effects included headache, fatigue, muscle pain and joint pain. Efficacy was based on a U.S. study of 266 people who received Ixchiq versus 96 on placebo. Protective antibody levels were shown in non-human primates that had received blood from people who had been vaccinated. The FDA reported that “almost all vaccine study participants achieved this antibody level.”

Nov 9:

Takeda is having a big week. One day after winning approval of colorectal cancer drug Fruzaqla, the Japanese pharma announced the FDA approval of Adzynma for congenital thrombotic thrombocytopenic purpura (cTTP). Adzynma is approved as either a preventive or on demand enzyme replacement therapy for adult and pediatric patients with cTTP.

A very rare, inherited disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making the ADAMTS13 enzyme that regulates blood clotting. Adzynma, a purified recombinant form of this enzyme, provides a replacement for the low levels of the deficient enzyme in patients with cTTP. Thursday’s approval marks the first treatment for this patient population.

Nov 8:

Wednesday turned out to be a big day at the FDA. Hours after greenlighting Eli Lilly’s obesity treatment Zepbound, the regulator approved Takeda’s Fruzaqla (fruquintinib) for previously treated patients with metastatic colorectal cancer. Fruzaqla targets the VEGF-1, -2 and -3 receptors, which together play a crucial role in the formation of the blood vessels that sustain tumors. The drug’s strong selectivity allows for higher doses while minimizing off-target effects for sustained inhibition of its targets.

The approval, which came 20 days before Takeda’s scheduled PDUFA date, makes Fruzaqla the “first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status,” according to the Japanese pharma.

Nov 8:

A new chapter opened Wednesday in the obesity treatment space as the FDA approved Eli Lilly’s Zepbound (tirzepatide) injection for chronic weight management in adults with obesity or excessive weight with at least one weight-related condition, such as hypertension, type 2 diabetes (T2D) or high cholesterol. Tirzepatide, the active ingredient in Zepbound, is already approved in Lilly’s Mounjaro for T2D.

Zepbound is the first and only approved treatment activating two incretin hormone receptors, GIP and GLP-1, to tackle an underlying cause of excess weight, according to Lilly’s announcement. Zepbound won approval on the strength of the Phase III SURMOUNT-1 and SURMOUNT-2 trials, where patients taking Lilly’s drug saw a “statistically significant” reduction in body weight compared to their placebo counterparts. The Indianapolis–based pharma intends to make Zepbound available in the U.S. by the end of this year.

Nov 1:

Erosive GERD patients have a new treatment option after the FDA greenlit Phathom Pharmaceuticals’ Voquezna (vonoprazan) tablets—which the gastrointestinal-focused company claims is the “first major innovation to the U.S. erosive GERD market in over 30 years.”

Approval of Voquezna—a novel, oral small molecule potassium-competitive acid blocker—was granted based on results of the Phase III PHALCON-EE study, which compared the drug to the acid-reducer lansoprazole in terms of healing and maintenance of erosive GERD and associated heartburn symptom relief. Voquezna met the study’s primary endpoint of non-inferiority after eight weeks of treatment and showed a healing rate of 93% compared to 85% for lansoprazole.

Nov 1:

Merck’s blockbuster Keytruda racked up another FDA approval Wednesday, as the company announced that the anti-PD-1 therapy is now authorized—in combination with gemcitabine and cisplatin—to treat patients with locally advanced unresectable or metastatic biliary tract cancer (BTC). BTC is the sixth U.S. GI cancer indication for Keytruda, which is also approved for esophageal or gastroesophageal cancer and HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma, among others.

Approval in BTC was granted based on results of the Phase III KEYNOTE-966 trial, where Keytruda elicited a “significant overall survival benefit” in this patient population versus chemotherapy alone, according to Merck’s announcement. Keytruda reduced the risk of death by 17% over chemo alone, with patients on the Keytruda plus chemo regimen surviving for a median 12.7 months versus 10.9 months for those receiving chemotherapy alone.

Oct 31:

Novartis picked up another indication for Cosentyx Tuesday as the FDA greenlit the IL-17A inhibitor to treat severe hidradenitis suppurativa, a painful, chronic skin condition characterized by boil-like lumps that typically burst into open wounds, leaving lasting scars. The approval marks the first new biologic treatment option for HS in almost a decade, according to Novartis’ announcement.

The approval was based on data from the SUNSHINE and SUNRISE studies, which together showed that Cosentyx-treated patients achieved significantly higher rates of clinical response compared to placebo. Safety was consistent with that observed in the plaque psoriasis trials, which Novartis said affirmed Cosentyx’s “differentiated safety profile.”

Oct 31:

J&J has new competition in the inflammatory disease space after the FDA approved Amgen’s Wezlana (ustekinumab-auub) for multiple such conditions. Wezlana is a biosimilar to and interchangeable with J&J’s Stelara and like Stelara, is approved to treat moderate to severe plaque psoriasis, active psoriatic arthritis, moderately to severely active Crohn’s disease and moderately to severely active ulcerative colitis. In the pediatric setting, Wezlana can be used to treat patients six and older with active psoriatic arthritis and moderate to severe plaque psoriasis.

On the safety side, Wezlana’s label lists infection as its most serious side effect and recommends discontinuing treatment in case of serious or clinically significant infections. As with other ustekinumab products, Wezlana carries the risk of malignancies, hypersensitivity reactions and non-infectious pneumonia.

Oct 27:

Coherus BioSciences and Shanghai Junshi Biosciences won approval Friday for Loqtorzi (toripalimab-tpzi)—in combination with cisplatin and gemcitabine—for the first-line treatment of metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC). This marks the first FDA-authorized treatment for NPC, a rare cancer. Loqtorzi is also approved as a monotherapy for adult patients with recurrent, unresectable or metastatic NPC with disease progression on or after platinum-containing chemotherapy.

The approval is based on the Phase III JUPITER-02 study where Loqtorzi combined with chemotherapy “significantly improved” progression-free survival, reducing the risk of disease progression or death by 48% compared to chemo alone. In terms of overall survival, treatment with Loqtorzi led to a 37% reduction in risk of death versus chemo alone.

Oct 27:

Roche’s Genentech can add a third indication to Vabysmo’s label after the FDA signed off on the bispecific antibody to treat macular edema—swelling—following retinal vein occlusion (RVO). Vabysmo is also approved for age-related macular degeneration and diabetic macular edema. It is the first and only bispecific antibody approved for the eye, according to Genentech’s announcement.

In the Phase III BALATON and COMINO studies, treatment with Vabysmo led to early and sustained improvement in vision in people with branch and central RVO, Genentech reported, which met the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to Regeneron’s Eylea (aflibercept). Vabysmo’s safety profile was consistent with earlier trials, though Genentech noted that information on “rare post-marketing reports of retinal vasculitis and/or retinal vascular occlusion” has been added to the drug’s label across indications.

Oct 26:

There’s a new treatment on the immediate horizon for patients with Duchenne muscular dystrophy after the FDA approved Santhera Pharmaceuticals’ vamorolone—now Agamree. An oral suspension drug, Agamree is indicated for DMD patients two years and older.

Patients with DMD are typically treated with glucocorticoid regimens, which, while effective at slowing muscle deterioration, come with significant side effects. Agamree elicits “differential effects” on glucocorticoid and mineralocorticoid receptors, modifying their downstream activities. This allows it to maintain the usual efficacy of glucocorticoid regimens while also achieving a better-tolerated side effect profile, according to Catalyst Pharmaceuticals, which will commercialize the drug in North America. Catalyst CEO Patrick McEnany touted Agamree’s “transformational potential” for DMD patients.

While Agamree’s label does not carry a boxed warning, it does list precautions regarding alterations in endocrine, cardiovascular and renal function, immunosuppression and ophthalmic side effects, among others.

Oct 26:

Eli Lilly won FDA approval Thursday for Omvoh (mirikizumab-mrkz) for the treatment of moderately to severely active ulcerative colitis (UC), marking the company’s first approved drug for a type of inflammatory bowel disease. Omvoh—which is the second new treatment option for UC patients approved this week after Celltrion’s Zymfentra—is the first to selectively target the p19 subunit of IL-23, which plays a role in inflammation related to the condition, according to Lilly.

Omvoh hit both primary and key secondary endpoints in its pivotal trials, including sustained clinical remission, and Lilly noted that the drug delivered “significant improvement in bowel urgency,” which patients reported as one of the most disruptive symptoms of UC.

Oct 24:

Servier on Tuesday won its fifth FDA approval for Tibsovo (ivosidenib tablets)—which also happens to be the first and only targeted therapy for patients with relapsed or refractory (R/R) myelodysplastic syndromes (MDS) with a susceptible IDH1 mutation. Tibsovo is joined in this indication by a companion diagnostic, Abbott Laboratories’ RealTime IDH1 Assay—also approved Tuesday—which physicians can use to identify patients eligible for Tibsovo treatment.

Tibosovo’s approval was supported by data from a pivotal Phase I open-label study of 18 patients from the target population, where the drug elicited a 38.9% complete remission rate after a median of 1.9 months. The objective response rate in the study was 83.3% and median overall survival was 35.7 months; median duration of complete response had not been reached at the time of the data cutoff.

Oct 23:

Ulcerative colitis (UC) and Crohn’s disease (CD) patients who have previously used intravenous infliximab now have a subcutaneous option after the FDA approved Celltrion’s Zymfentra on Monday. Zymfentra is the first and only subcutaneous version of infliximab for the maintenance treatment of adults with inflammatory bowel disease, according to Celltrion.

Infliximab, approved in 1998 and marketed by J&J’s Janssen under the name Remicade, is a monoclonal antibody that works by blocking the pro-inflammatory cytokine TNF-alpha. In the Phase III LIBERTY-UC and LIBERTY-CD studies, Zymfentra elicited higher rates of clinical remission than placebo in patients with moderate-to-severe UC and CD, respectively, following induction with intravenous infliximab.

Oct 20:

Nearly two years ago, the FDA approved BioMarin’s Voxzogo (vosoritide) as the first therapy for achondroplasia, a rare genetic disorder that causes the most common form of dwarfism—fueling ongoing controversy within the dwarfism community about such treatments. That approval was for children five and older. On Friday, the FDA expanded Voxzogo’s label to include kids under five.

Voxzogo is designed to increase linear growth in pediatric patients with achondroplasia who have open growth plates. Friday’s supplemental approval enables Voxzogo to be prescribed to all children with open growth plates, the California–based company said in its announcement. Accelerated approval was granted based on an “improvement in annualized growth velocity,” according to BioMarin.

Oct 20:

Sanofi and Regeneron suffered a rare defeat with Dupixent this weekend as the FDA issued a Complete Response Letter to their supplemental Biologics License Application in chronic spontaneous urticaria, an inflammatory skin condition that leads to debilitating hives.

In its rejection, the regulator said additional efficacy data are required to support an approval. No safety or manufacturing issues were noted. Sanofi and Regeneron are running an ongoing study which they expect to provide this data, the pharma partners said in their announcement. Results of this study are expected late next year.

Oct 20:

Pfizer’s vaccines business got a boost on Friday as the FDA greenlit Penbraya as the “first and only vaccine” covering the five most common serogroups causing meningococcal disease in adolescents and young adults 10 to 25 years of age.

Annaliesa Anderson, Pfizer’s senior vice president and head of vaccine research and development, touted the benefits of Penbraya—which combines components from two other meningococcal vaccines, Trumenba and Nimenrix—saying the new vaccine “has the potential to protect more adolescents and young adults from this severe and unpredictable disease by providing the broadest meningococcal coverage in the fewest shots.”

Approval for Penbraya was granted based on positive results from Phase II and III trials, including a Phase III study that sought to determine the immunologic noninferiority of the vaccine against meningococcal vaccines currently available in the U.S. in terms of safety, tolerability and immunogenicity.

Oct 18:

The gene therapy space celebrated another first on Wednesday, as the FDA cleared the way for the first-ever investigational in vivo CRISPR-based gene editing therapy to enter late-stage development. Intellia’s NTLA-2001, being developed along with partner Regeneron for the genetic liver disease transthyretin (ATTR) amyloidosis cardiomyopathy, is expected to enter Phase III by the end of this year, Intellia announced.

Intellia and Regeneron made history in June 2021 with NTLA-2001 when they announced positive early data from the first-ever patients to have their DNA edited with an in vivo CRISPR/Cas9 therapy delivered systemically.

Oct 18:

It’s been a charmed week for UCB, which on Wednesday announced its second straight approval, this one for bimekizumab—now Bimzelx—for adults with moderate-to-severe plaque psoriasis. Bimzelx is the first FDA-approved plaque psoriasis medication to inhibit both the IL-17A and IL-17F cytokines, each of which is a key player in the inflammatory cascade.

Bimzelx’s road to approval has not been without its trials. Its regulatory path was first interrupted by the COVID-19 pandemic; UCB reported in October 2021 that the FDA had to delay its verdict on the drug’s Biologics License Application because it was unable to conduct on-site facility inspections due to travel restrictions. Then, in May 2022, UCB received a Complete Response Letter that cited “pre-approval inspection observations” the company would need to address before Bimzelx could be approved.

Oct 17:

A long road for Ardelyx’s tenapanor ended in victory Tuesday as the FDA approved the phosphate absorption inhibitor to reduce serum phosphorus levels in patients with chronic kidney disease (CKD). Specifically, tenapanor—to be marketed as Xphozah—is intended for CKD patients on dialysis who have previously shown an inadequate response to phosphate binders or who are otherwise intolerant of such therapies.

Xphozah’s path has not been without challenges. In July 2021, the FDA issued Ardelyx a Complete Response Letter in which it stated that the molecule’s effect size was “small and of unclear clinical significance.” Then, in November 2022, an FDA advisory committee voted 9-4 that Xphozah’s benefits outweighed its risks. Ardelyx’s revamped New Drug Application included data from the Phase III PHREEDOM, BLOCK and AMPLIFY studies, all of which met their primary efficacy endpoints.

Oct 17:

The FDA signed off on the first once-daily subcutaneous targeted C5 complement inhibitor for generalized myasthenia gravis (gMG) in the form of UCB’s zilucoplan, now Zilbrysq. It is also the only once-daily target therapy for gMG that patients can self-administer for the rare autoimmune disease, according to UCB’s announcement.

Zilbrysq won approval based on the Phase III RAISE study, which assessed the therapy in adults with anti-acetylcholine receptor antibody-positive gMG. Zilbrysq demonstrated “rapid, consistent and statistically significant benefits” after 12 weeks in various patient- and clinician-reported outcomes, according to UCB. Zilbrysq’s label comes with a boxed warning for serious meningococcal infections, as “life-threatening and fatal episodes” of these infections have been observed with other C5 complement inhibitors. UCB noted that there have been no life-threatening or fatal meningococcal infections in patients treated with Zilbrysq to date.

Oct 16:

Patients with early-stage non-small cell lung cancer (NSCLC) can now rely on just one drug—Merck’s Keytruda—before and after surgery, in addition to chemotherapy during pre-surgical treatment, after the FDA approved the blockbuster Monday as a continuous regimen for these patients.

Approval was based on data from the KEYNOTE-671 trial, where Keytruda plus chemotherapy showed an advantage over placebo plus chemo in both overall survival and investigator-assessed event-free survival. Neither indicator was reached for patients taking Keytruda, while those on placebo survived an average of 52.4 months, with an event-free survival time of 17 months.

The approval is the sixth for Keytruda in NSCLC, according to Merck, which made the announcement Monday evening.

Oct 14:

Bristol Myers Squibb can add another adjuvant indication to Opdivo’s label after the FDA approved the immunotherapy blockbuster to treat patients 12 and older with completely resected stage IIB or IIC melanoma. According to BMS’ announcement, Opdivo is the only PD-1 inhibitor indicated for both this patient population and for stage III and stage IV completely resected melanoma.

The approval was granted based on the results of the Phase III CheckMate -76K trial, where Opdivo reduced the risk of recurrence, new primary melanoma or death by 58% compared to placebo. After one year of treatment with Opdivo, recurrence-free survival was 89% compared with 79% for placebo.

Oct 13:

Pfizer’s $6.7 billion acquisition of Arena Pharmaceuticals paid off in a big way Friday as the FDA approved etrasimod to treat moderate to severe ulcerative colitis (UC). Etrasimod—which will henceforth be known as Velsipity in this indication—was the centerpiece of Pfizer’s 2021 pick-up of Arena.

The approval was based on results from Pfizer’s ELEVATE UC Phase III registrational program. In May 2022, Pfizer announced data from two pivotal trials of the selective sphingosine 1-phospate (S1P) receptor modulator. The 52-week ELEVATE UC 52 study showed 27% clinical remission in patients taking Velsipity versus 7.4% in those who were part of the placebo group at week 12, and 32.1% clinical remission compared with 6.7% at week 52. In ELEVATE UC 12, clinical remission was 24.8% in those who received Velsipity compared to 15.2% in those who were given a placebo.

Velsipity’s label notes that the drug may increase the risk of infections and cause bradyarrhythmia and atrioventricular conduction delays. Velsipity showed a “favorable safety profile” across the Phase III program, according to Pfizer’s approval announcement.

Oct 12:

Alvotech has been iced out by “deficiencies” at the company’s manufacturing facility in Reykjavik, Iceland, which the FDA cited in a Complete Response Letter it issued in response to Alvotech’s bid for approval of AVT04. The company was proposing AVT04 as a biosimilar to J&J’s Stelara (ustekinumab), which binds to binds to two cytokines, IL-12 and IL-23, that are involved in inflammatory and immune responses.

The deficiencies, which were identified via an FDA inspection of the Reykjavik facility that concluded in March, “must be satisfactorily resolved before the application can be approved,” Alvotech noted in its announcement. No other issues were flagged, according to Alvotech. The company plans to file a resubmission “shortly,” which it said would trigger another six-month review cycle and a new target action date.

Oct 12:

PepGen’s Phase I myotonic dystrophy type 1 (DM1) trial is cleared to begin in the U.S. after the FDA lifted a full clinical hold it had placed on the Investigational New Drug Application for PGN-EDODM1. PepGen announced the hold in May but did not provide details as to the reason for it.

“We have worked closely with the FDA to resolve their questions expeditiously and are pleased that the clinical hold on our DM1 program in the United States has been lifted,” PepGen president and CEO James McArthur said in Thursday’s press release.

The Phase I trial will study target dose levels of 5 mg/kg, 10 mg/kg and 20 mg/kg of PGN-EDODM1, with safety, transcript splicing and clinical outcome measures data from the 5 mg/kg cohort expected in 2024.

Oct 12:

The FDA greenlit Pfizer’s Braftovi (encorafenib)/Mektovi (binimetinib) combination for the treatment of adult patients with non-small cell lung cancer (NSCLC) with a BRAF V600E mutation as confirmed by an FDA-approved test.

“BRAF V600E mutations identify a unique subtype of metastatic non-small cell lung cancer that presents an actionable biomarker that precision medicines like [the Braftovi/ Mektovi] combination therapy can help address,” said Gregory Riely, vice chair of clinical research in the Department of Medicine at Memorial Sloan Kettering Cancer Center and a PHAROS investigator, in a prepared statement.

The approval was based on data from the ongoing Phase II PHAROS clinical trial, which is studying the combination in both treatment-naïve and previously treated patients with BRAF V600E-mutant metastatic NSCLC. The trial met the major efficacy outcome measures of objective response rate and duration of response in both treatment groups, according to Pfizer’s announcement.

Oct 9:

In a relatively rare move, the FDA went against the recommendation of one of its advisory committees, rejecting a bid by Alnylam to expand Onpattro (patisiran)’s label to cardiomyopathy of transthyretin-mediated amyloidosis. In September, the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in patisiran’s favor, but on Monday, Alnylam announced that the regulator had issued a Complete Response Letter in response to its supplemental New Drug Application in this indication. In the CRL, the FDA said Alnylam had not provided enough evidence of the therapy’s benefit.

In the same announcement, Alnylam said it would no longer work toward an expanded label for the siRNA therapeutic in cardiomyopathy of ATTR amyloidosis in the U.S.

Oct 6:

People with a range of rheumatic diseases have a new treatment delivery option after the FDA approved an intravenous formulation of Novartis’ IL-17A antagonist Cosentyx (secukinumab). The new authorization covers existing indications for Cosentyx, including ankylosing spondylitis (AS), psoriatic arthritis (PsA) and non-radiographic axial spondyloarthritis (nr-axSpA). A fully human monoclonal antibody, Cosentyx, which works by targeting and inhibiting the IL-17A cytokine, thereby suppressing the inflammatory pathway, was previously only authorized as a subcutaneous treatment in these indications.

The IV route of administration will improve treatment access for “a significant portion of patients” with AS, PsA and nr-axSpA who are not comfortable with self-injections or simply prefer to receive treatments in their healthcare provider’s office, said Philip Mease, clinical professor at the University of Washington School of Medicine, in a prepared statement.

Oct 6:

Arcutis Biotherapeutics is adding another indication for its psoriasis cream, Zoryve, after the FDA approved the company’s supplemental New Drug Application (sNDA) to expand the treatment for children ages 6 to 11. Zoryve is a once-daily, steroid-free cream, an option that Adelaide A. Hebert, professor and chief of pediatric dermatology at McGovern Medical School at UTHealth Houston and Children’s Memorial Hermann, said in a statement is “especially needed for managing plaque psoriasis in younger children.”

Zoryve won approval in July 2022 to treat patients psoriasis patients 12 years and older.

Oct 5:

Amgen has “interpretation” issues—of the confirmatory study variety, at least according to an FDA advisory committee that voted against converting the accelerated approval of its oral G12C KRAS inhibitor Lumakras (sotorasib) to full approval in non-small cell lung cancer (NSCLC). In a 10-2 vote Thursday, the Oncologic Drugs Advisory Committee determined that progression-free survival (PFS) data from the Phase III confirmatory CodeBreaK 200 study could not be reliably interpreted. The advisers pointed to the high number of study dropouts, the small sample size and potentially biased behavior of the trial’s investigators.

Panelist Mark Conaway, a professor at the Division of Translational Research and Applied Statistics at the University of Virginia School of Medicine, said that in CodeBreaK 200, there were “. . . a large number of issues that cloud the interpretation of a small observed effect.”

Lumakras was approved under the FDA’s accelerated approval pathway in May 2021.

Oct 4:

The FDA’s Oncologic Drugs Advisory Committee voted 14-6 in favor of US WorldMeds’ neuroblastoma drug Wednesday—even without the benefit of a randomized controlled trial. The external advisers ultimately determined there was enough evidence to conclude that eflornithine hydrochloride (DFMO) boosts event-free survival in pediatric patients with high-risk neuroblastoma. In its application, US WorldMeds submitted data from a prospective Phase II, multicenter, open-label, single-arm study of DFMO maintenance treatment of patients who had completed multiagent and multimodality therapy.

While voting “yes,” Christopher Lieu, committee chairperson and director of the Gastrointestinal Medical Oncology Program at the University of Colorado, acknowledged that a positive decision could lead to a “slippery slope” in terms of the degree of clinical evidence that will be required of future drug applications in similar rare and difficult-to-study indications.

Oct 3:

The latest updated COVID-19 vaccine will soon be available in U.S. retail pharmacies and physicians’ offices after the FDA approved the 2023-2024 formulation of Novavax’s adjuvanted shot, NVX-CoV2601 under its Emergency Use Authorization pathway. The vaccine is also now included in the CDC’s recommendations, initially issued September 12. Doses of the updated vaccine will be available across the U.S. “in the coming days,” Novavax announced Tuesday. The EUA is for individuals 12 years and older.

Oct 2:

Rocket Pharmaceuticals has lift-off in Leukocyte Adhesion Deficiency-I (LAD-I). The Cranbury, New Jersey–based biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections. Without an allogeneic hematopoietic stem cell transplant, most patients do not live past childhood. Kinnari Patel, Rocket’s president and chief operating officer, noted that this treatment option “has substantial morbidity and mortality and may not be available in time for these children.”

RP-L201 holds the FDA’s Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, Fast Track and Orphan Drug designations.

Oct 2:

Adults and kids nine and older with primary hyperoxaluria type 1 (PH1)—a rare genetic disease—have a new treatment option after the FDA approved Novo Nordisk’s Rivfloza. The approval for Novo’s first RNAi therapy was based on data from the pivotal Phase II PHYOXTM2 trial and interim data from the ongoing Phase III PHYOXTM3 extension study. Rivfloza met the primary endpoint in PHYOXTM2 as patients in the treatment group saw a marked reduction from baseline in 24-hour-urinary oxalate (Uox) excretion from Day 90 to Day 180, according to Novo’s press release announcing the decision.

Primary hyperoxaluria causes an overproduction of oxalate by the liver. PH1, the most prevalent and severe subtype, primarily affects the kidneys and can lead to progressive kidney damage. It is estimated to affect more than 2,000 people in the U.S., according to Novo.

“RNA interference is a proven treatment approach for individuals with PH1,” David S. Goldfarb, clinical chief of the nephrology division at NYU Langone Medical Center and professor of medicine and physiology at NYU Grossman School of Medicine, said in the same press release. “With the approval of Rivfloza, we now have a novel treatment that lowers oxalate production safely and effectively.”

Oct 2:

Eli Lilly will have to wait a little longer to see a payoff from lebrikizumab, which it picked up in its $1.1 billion acquisition of Dermira in January 2020. On Monday, the FDA issued a Complete Response Letter for lebrikizumab due to inspection findings at a third-party manufacturer. Lebrikizumab was being proposed to treat moderate-to-severe atopic dermatitis (eczema). The CRL did not cite any concerns about Lilly’s clinical data package, safety or label for lebrikizumab, according to a company press release.

In the statement, Patrik Jonsson, executive vice president, president of Lilly Immunology and Lilly USA, and chief customer officer, expressed confidence in lebrikizumab’s potential and said the company would continue to work closely with the unnamed third-party manufacturer and FDA in order to bring the drug to patients.

Sept 29:

On Friday, the FDA approved Biogen’s Tofidence as the first biosimilar to Roche’s Actemra (tocilizumab) in the U.S. Tofidence is authorized to treat moderately to severely active rheumatoid arthritis (RA), polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.

The approval was based on a three-arm, parallel Phase I study that compared the pharmacokinetics, safety and immunogenicity of Tofidence to reference tocilizumab in healthy volunteers; a Phase III study compared the two products in RA not sufficiently controlled by methotrexate in order to establish equivalent efficacy and comparable pharmacokinetic, safety and immunogenicity profiles.

To illustrate the value of a biosimilar in this space, Biogen referenced in its press release a report by the Association for Accessible Medicines which states that spending on therapies for autoimmune diseases has consistently increased by 10% to 25% each year over the past decade. “The entry of new biosimilar competition in 2023 and 2024 is projected to dramatically reduce this trend,” according to the report.

Sept 28:

Adults with late-onset Pompe disease (LOPD) have a new treatment option after the FDA approved Amicus Therapeutics’ Pombiliti (cipaglucosidase alfa-atga) plus Opfolda (miglustat). The treatment—the first and only two-component therapy for patients with LOPD—is specifically for individuals who are not their current enzyme replacement therapy. LOPD is a life-threatening lysosomal disorder caused by a deficiency of acid alpha-glucosidase (GAA). Pombiliti is a recombinant human GAA enzyme that is taken up into the muscle cells while Opfolda is an enzyme-stabilizer designed to stabilize Pombiliti in the blood.

Amicus intends to launch the treatment in the U.S. “immediately.”

The depression treatment space has had a landmark year with the August approval of Biogen and Sage Therapeutics’ Zurzuvae as the first pill for postpartum depression—though the drug was rejected for major depressive disorder (MDD). On Thursday, the FDA approved a new therapy for that indication in Fabre-Kramer Pharmaceuticals’ Exxua (gepirone hydrochloride tablets).

The approval of Exxua—which Houston–based Fabre-Kramer states is the “first and only approved” antidepressant for adults with MDD that works through the selective agonism of 5HT1a receptors to modulate serotonin throughout the central nervous system—was hard won. The privately held company first filed for approval of the drug in September 1999 but the New Drug Application was rejected by the FDA in 2002. Exxua’s label includes a boxed warning for suicidal thoughts and behaviors in children and young adults, for whom the drug is not approved. It does not, Fabre-Kramer notes, carry precautions for sexual dysfunction and weight gain. The company plans to launch Exxua in early 2024.

Sept 27:

Takeda on Wednesday won approval for a subcutaneous (SC) form of Entyvio as a maintenance therapy for adults with moderately to severely active ulcerative colitis (UC) following induction therapy with intravenous Entyvio. The SC treatment is expected to be available in the U.S. by the end of October as a single-dose pre-filled pen.

Bruce Sands, chief of the Dr. Henry D. Janowitz Division of Gastroenterology at the Icahn School of Medicine at Mount Sinai, lauded the SC approval, saying he appreciates being able to provide “appropriate UC patients a choice of how they receive their maintenance therapy.”

Entyvio, which is also under FDA review for severely active Crohn’s disease, was first approved in 2014.

After a long day of presentations and deliberations, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted overwhelmingly to reject BrainStorm Cell Therapeutics’ experimental amyotrophic lateral sclerosis (ALS) therapy NurOwn. With a tally of 17-1, the advisory committee (adcomm) determined that there was not substantial evidence that NurOwn, a cell therapy made up of mesenchymal stromal cells secreting neurotrophic factors (MSC-NTF), was an effective treatment for patients with mild to moderate ALS.

The outcome was not a surprise after FDA briefing documents released on Monday took issue with NurOwn’s manufacturing plan and said the company failed to demonstrate substantial evidence of efficacy in its Biologics License Application. BrainStorm filed the BLA using the FDA’s File Over Protest procedure after the regulator issued a Refuse to File letter in November 2022.

Ocuphire and Viatris won approval Wednesday for Ryzumvi (phentolamine ophthalmic solution) for the reversal of pharmacologically-induced pupil dilation. While the topical drug does not come with a boxed warning, its label does carry warnings for uveitis and other common adverse effects of treatment, including site discomfort and conjunctival hyperemia. The partners intend to launch Ryzvumi in the U.S. in the first half of 2024.

Artificial dilation can last from six to 24 hours, creating barriers for some patients. “Our hope is that by addressing patient dilation barriers, we’re empowering eye care professionals to broaden exam availability, leading to enhanced eye health outcomes,” Viatris Eye Care division president Jeffrey Nau said in a prepared statement.

Sept 25:

Coherus BioSciences just can’t seem to catch a regulatory break this week. The company announced Monday that Udenyca Onbody, its updated biosimilar to Amgen’s Neulasta, has been rejected by the FDA due to an ongoing issue at a third-party filler. The Complete Response Letter did not note any issues with the biosimilar’s clinical efficacy or safety, trial design, labeling, drug substance manufacturing or device design or manufacturing, Coherus stated in a press release. Neulasta is used to prevent neutropenia, a lack of white blood cells caused by chemotherapy.

In the same announcement, Coherus reported that in the process of inspecting three sites in China enrolling patients for two pivotal trials of its PD-1 antibody toripalimab in recurrent nasopharyngeal carcinoma (NPC), the FDA identified one “observation”. Toripalimab is being proposed as a first-line, second-line or greater treatment for metastatic or recurrent NPC. Coherus said the observation—communicated in an FDA Form 483—is readily addressable and the company “continues to anticipate potential approval for toripalimab by year end 2023.”

Coherus and its partner Junshi Biosciences received a CRL for toripalimab in this indication requesting a “quality process change” in May 2022.

Sept 25:

Canadian biopharma company Appili Therapeutics announced the FDA approval of Likmez—formerly ATI-1501—a liquid oral reformulation of the antibiotic metronidazole. With more than 10 million prescriptions filled annually in the U.S., metronidazole is a front-line oral antibiotic used commonly for parasitic and anaerobic bacterial infections, according to Appili’s website. Prior to the approval of Likmez, metronidazole was only available in tablet form, which posed therapeutic barriers for very young patients and those who have difficulties swallowing, especially the elderly. New York–based Saptalis Pharmaceuticals will commercialize Likmez in the U.S.

Located in Halifax, Nova Scotia, Appili is developing novel approaches to treat infectious diseases. Also in the pipeline are a topical product for the disfiguring skin infection cutaneous leishmaniasis and a live attenuated vaccine against aerosolized bacteria Francisella tularensis, which the National Institutes of Health defines as a Category A pathogen.

Sept 22:

Nearly two months after winning approval in the EU for Jardiance in chronic kidney disease (CKD), Eli Lilly and Boehringer Ingelheim can add CKD to the SGLT2 inhibitor’s label stateside too. The FDA nod, which the companies announced on Friday, is specifically for the reduction of risk of sustained decline in estimated glomerular filtration rate (eGFR), end-stage kidney disease and cardiovascular death and hospitalization in adults with CKD at risk of progression.

The ”meaningful benefits” shown by Jardiance in the Phase III EMPA-KIDNEY trial “are welcome news for adults living with CKD in [the U.S.],” Katherine Tuttle, executive director for research at Providence Inland Northwest Health, regional principal investigator for the Institute of Translational Health Sciences, professor of medicine at the University of Washington, and a steering committee member for the trial, said in a prepared statement.

In EMPA-KIDNEY, which enrolled more than 6,600 patients, Jardiance combined with standard of care elicited a 28% relative risk reduction in kidney disease progression, defined as end-stage kidney disease, or cardiovascular death compared with placebo plus standard of care. Jardiance also met the trial’s secondary endpoint, showing a 14% reduction in the risk of first and recurrent hospitalization compared to placebo, a result that was deemed significant.

Sept 21:

In a setback for Intarcia Therapeutics, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously that the benefits of its drug-device combination for Type 2 diabetes, ITCA 650, did not outweigh the risks. The panel cited the high rate of acute kidney injury (AKI) in patients assigned to the treatment group, which it said could potentially be attributed to ITCA 650. The advisors concluded that there is a lot of uncertainty surrounding ITCA 650’s safety profile and that additional data are needed, which they suggested Intarcia could gather in larger trials.

Intarcia has walked a troubled regulatory path with ITCA 650, suffering two FDA rejections for ITCA 650. The first Complete Response Letter, in which the regulator cited manufacturing issues, came in 2017, and the FDA issued a second CRL in March 2022, flagging signals of AKI.

Sept 20:

The FDA surprised ARS Pharmaceuticals—if the tone of the company’s press release is any indication—by issuing a Complete Response Letter for neffy, an epinephrine nasal spray, in type 1 allergic reactions, including anaphylaxis. The regulator went against the recommendation of its Pulmonary-Allergy Drugs Advisory Committee (PADAC), which in May voted 16-6 in favor of the treatment in adults and 17-5 in kids 18 years and younger.

In its CRL announcement, ARS said it had aligned with the FDA on physician labeling and post-market requirements, which included a repeat-dose study of neffy under allergen-induced allergic rhinitis conditions. The agency is now requesting a repeat-dose study be completed before it will approve neffy. FDA is also asking that ARS submit additional information on testing for nitrosamine impurities based on new draft guidance that was issued after
neffy’s New Drug Application was submitted in October 2022.

ARS intends to submit a Formal Dispute Resolution Request to appeal the CRL. The company plans to resubmit the application for neffy in the first half of next year and anticipates another FDA action date in the second half of 2024.

Sept 16:

After a three-month delay, the FDA handed GSK a victory Friday, approving momelotinib—to be henceforth known as Ojjaara—for the treatment of myelofibrosis with anemia. Ojjaara’s label is for adult patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis who suffer from anemia. The JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor is the only approved drug for both newly diagnosed and previously treated myelofibrosis patients with anemia to address key disease manifestations including anemia, constitutional symptoms and enlarged spleen, according to GSK’s press release.

“The vast majority of myelofibrosis patients eventually develop anemia, causing them to discontinue treatments and require transfusions,” said Nina Mojas, senior vice president of oncology global product strategy at GSK in the same press release.

By silencing the JAK1 and JAK2 pathways, momelotinib alleviates symptoms of splenomegaly and elicits improvements in patients’ constitution.

Sept 14:

Iovance Biotherapeutics just can’t catch a break when it comes to the regulatory review of Lifileucel, its tumor-infiltrating lymphocyte (TIL) therapy for advanced melanoma. Originally anticipating an FDA decision on or before November 25, the San Carlos, Calif–based company will now have to wait until 2024 due to “resource constraints” on behalf of the regulator, Iovance announced Thursday.

Nearly three years ago, in October 2020, Iovance’s planned Biologics License Application was pushed back when the FDA requested additional data on Lifileucel’s potency assays. Then, in May 2021, the regulator requested more data on the potency assays. In March 2023, Iovance finally completed a rolling BLA submission, which the FDA accepted in May, setting the late November action date.

The new action date is February 24, 2024. Successful facility inspections have been completed and there have been no major review issues, according to Iovance’s press release. The FDA’s Center for Biologics Evaluation and Research is expecting an influx of cell and gene therapy applications, and in March 2023 established a new super office, the Office of Therapeutic Products, in an attempt to keep up with the demand.

Sept 14:

After announcing Tuesday that its CRF1 antagonist, crinecerfont, met the primary efficacy endpoint in the Phase III CAHtalyst Adult Study for classic congenital adrenal hyperplasia, Neurocrine Biosciences reported good news on the neuro front. The FDA has accepted the San Diego–based company’s New Drug Application for Ingrezza (valbenazine) oral granules, a new sprinkle formulation of the capsules, which are approved to treat tardive dyskinesia and chorea associated with Huntington’s disease. The oral granule capsules—which come in 40 mg, 60 mg and 80 mg doses—are intended to be opened and sprinkled on soft foods prior to administration. The FDA has set an action date of April 30, 2024.

Sept 14:

Hemogenyx Pharmaceuticals could be headed to the clinic with its CAR-T therapy HEMO-CAR-T after announcing that the FDA has accepted its plan to address concerns that led the regulator to place it on a clinical hold in July 2023. In the review letter, the FDA cited a “splicing deficiency” that arises during the production of the lentivirus used to create the CAR-T cells for HEMO-CAR-T.

Hemogenyx responded in August with a “detailed plan” supported by laboratory tests to address the FDA’s comments, according to a press release issued Thursday. The company will resubmit the Investigational New Drug Application “as expeditiously as possible in order to move forward with clinical trials,” Hemogenyx CEO Vladislav Sandler said in the same press release. HEMO-CAR-T is being developed to treat acute myeloid leukemia.

Sept 13:

Alnylam won support from an FDA advisory committee Wednesday for patisiran as a treatment for adults with cardiomyopathy induced by transthyretin amyloidosis (ATTR-CM). The FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 that patisiran’s benefits outweigh its risks in this indication, despite concerns expressed in FDA briefing documents prior to the adcomm meeting.

While Alnylam’s Phase III APOLLO-B study hit both key primary and secondary endpoints, the FDA stated in the documents that “the effects of patisiran compared to placebo on both endpoints were small, of questionable clinical meaningfulness, and may not be detectable by patients.” Major discussion points during the adcomm meeting included whether these key endpoints were appropriate to ascertain clinical meaningfulness in the ATTR-CM population, and panelists expressed varying opinions on whether the magnitude of the benefit seen in trial data was clinically meaningful. The FDA is set to render its decision on patisiran in ATTR-CM on or before Oct. 8, 2023.

Sept 13:

The FDA accepted for review Takeda’s Biologics License Application for a subcutaneous (SC) formulation of Entyvio for moderately to severely active Crohn’s disease following induction therapy with intravenous Entyvio. Takeda is supporting the BLA with data from the pivotal VISIBLE 2 Phase III trial, in which 409 adult patients experienced a clinical response—defined as a decrease of 70 points or more in Crohn’s Disease Activity Index (CDAI) score from baseline—following two doses of open-label IV Entyvio.

This marks the second BLA for SC Entyvio under FDA review, as the regulator accepted a BLA resubmission for the formulation for maintenance therapy in adults with moderately to severely active ulcerative colitis after IV Entyvio induction therapy in April 2023. The second submission was in response to a Complete Response Letter issued by the FDA in December 2019.

Sept 13:

Madrigal Pharmaceuticals—widely hailed as the leader in the nonalcoholic steatohepatitis (NASH) space—announced Wednesday that the FDA has accepted and granted priority review for its New Drug Application for resmetirom. The regulator set an action date of March 14, 2024, by which it must decide whether or not to approve the once daily, oral, thyroid hormone receptor (THR)-β selective agonist to treat NASH with liver fibrosis. In more good news for Madrigal, the FDA noted that it is not planning to hold an advisory committee meeting for the application.

The Pennsylvania-based company is supporting the NDA with a clinical development program that consists of 18 studies, including four Phase III trials. In December, the company’s shares rocketed 209% when it announced that the candidate had hit both primary endpoints and a secondary endpoint in the Phase III MAESTRO-NASH biopsy trial. Madrigal is requesting approval of resmetirom under the FDA’s accelerated approval pathway.

Sept 11:

As the leaves begin to turn across the U.S., the first updated COVID-19 booster shots are here. The FDA on Monday approved shots from both Pfizer-BioNTech and Moderna, which are specifically formulated to provide protection against circulating Omicron-related variants, including subvariant XBB.1.5, which the regulator had recommended the shots be updated to cover. The new boosters are approved for individuals 12 and above, with Emergency Use Authorization given for children aged six months to 11 years. On Tuesday, the Centers for Disease Control and Prevention signed off on the boosters and recommended that everyone 6 months and above receive them as CDC data indicate that hospitalizations are ticking up across the country.

Sept 11:

Israel–based BioLineRx won FDA approval for Aphexda (motixafortide), a stem cell mobilization agent for patients with multiple myeloma scheduled for autologous stem cell transplantation (ASCT). Aphexda was approved in combination with filgrastim (G-CSF), a granulocyte colony-stimulating factor that stimulates the growth of neutrophils. The FDA nod is based on the Phase III GENESIS trial, which compared Aphexda plus filgrastim against placebo plus filgrastim. In a single apheresis session, one round of add-on Aphexda mobilized the optimal number of stem cells in around 90% of patients; just 10% of patients who received granulocyte-CSF with placebo reached a similar level of stem cell mobilization.

Aphexda is the first approved therapeutic for BioLineRx, which also has offices in Waltham, Mass.

Sept 8:

At the end of July, Citius Pharmaceuticals received a Complete Response Letter from the FDA pertaining to its Biologics License Application for Lymphir, an engineered IL-2-diphtheria toxin fusion protein intended to treat relapsed or refractory cutaneous T-cell lymphoma. Friday, the Cranford, New Jersey–based biopharma announced that the FDA has agreed with its plans to address the “requirements” outlined in the CRL. In its decision, the regulator told Citius it would need to incorporate enhanced product testing and additional controls that were agreed to during the market application review.

Citius is “encouraged by the constructive engagement with the FDA,” Leonard Mazur, the company’s chairman and CEO, said in a statement on Friday, adding that Citius plans to complete the “remediation activities” by the end of 2023 and file a resubmission early in 2024.

Sept 6:

AstraZeneca will have to wait to add another indication to the label of blockbuster drug Ultomiris, as the FDA rejected the company’s supplemental application in neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease particularly affecting the optic nerve.

In its complete response letter, the regulator requested that AstraZeneca improve Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) strategy to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment with the C5 complement inhibitor. The FDA did not cite any issues with Ultomiris’ efficacy or safety data from the Phase III CHAMPION-NMOSD trial.

Sept 5:

The FDA gave tentative approval to Viatris’ abacavir 60 mg/dolutegravir 5 mg/lamivudine 30 mg tablets to treat pediatric HIV patients. The nod came through President Joe Biden’s Emergency Plan for AIDS Relief (PEPFAR) program, and indicates that the formulation meets the FDA’s quality, safety and efficacy standards, according to Viatris’ press release. The Pennsylvania–based company has signed a licensing agreement for pediatric dolutegravir with the Medicines Patent Pool and a development agreement with ViiV Healthcare and the Clinton Health Access Initiative to produce and distribute the fixed-dose combination of abacavir/ dolutegravir/lamivudine.

While children account for 4% of people living with HIV, they made up 13% of AIDS-related deaths in 2022 due to a lack of antiretroviral therapy, Viatris stated in the same press release.

Aug 30:

Outlook Therapeutics lost nearly three-quarters of its market share after the FDA declined to approve ONS-5010, an investigational ophthalmic formulation of the cancer drug bevacizumab being developed for wet age-related macular degeneration (AMD). In its complete response letter (CRL), the FDA cited “several CMC issues, open observations from pre-approval manufacturing inspections and a lack of substantial evidence,” according to Outlook.

The New Jersey–based biopharma intends to request a formal meeting with the FDA to “further understand the BLA deficiencies and how best to resolve them,” Russell Trenary, the company’s president and CEO, said in a statement.

ONS-5010 is an ophthalmic formulation of bevacizumab, a VEGF inhibitor sold by Roche’s Genentech as Avastin for several types of cancer. In eye diseases, it prevents the interaction of VEGF with its receptors on the surface of endothelial cells, reducing endothelial cell proliferation, vascular leakage and the formation of new blood vessels in the retina.

Aug 29:

In what could be a major boon to its $4 billion–plus sales aspirations, BMS scored first-line status for Reblozyl in adult patients with anemia in low- to intermediate-risk myelodysplastic syndromes (MDS) who may require blood transfusions. First approved to treat MDS-related anemia in 2020 in patients who failed to respond to an erythropoiesis-stimulating (ESA) agent—the established front-line treatment—the treatment will now be available to patients who have not received ESA therapy.

The first-line nod was backed by interim data from the Phase III COMMANDS trial, which pitted Reblozyl against the ESA standard of care treatment, Amgen and Johnson & Johnson’s Epogen/Procrit. More than 58% of patients receiving BMS’s drug achieved transfusion independence versus 31% of the comparator. And while Reblozyl was previously only available to patients exhibiting ring sideroblasts—blood cells with iron deposits circling the nucleus—it is now approved to treat those with and without this common symptom.

Aug 24:

Sandoz, the generics and biosimilars arm of Novartis, has earned FDA approval for the first biosimilar drug to treat multiple sclerosis. Tyruko (natalizumab-sztn) is a biosimilar of Biogen’s treatment Tysabri (natalizumab), a monotherapy used to treat adults with relapsing forms of MS. The biosimilar is also approved to treat adults with Crohn’s disease, another approved indication for Tysabri. Both drugs come with the risk of opportunistic viral infection progressive multifocal leukoencephalopathy and will carry a boxed warning about the potentially severe complication.

Aug 24:

Gilead Sciences’ antiviral drug Veklury (remdesivir) earned a label expansion from the FDA, now cleared for COVID-19 patients with all stages of liver disease. The dose will be the same as it was for its originally approved uses in adult and pediatric patients with mild to moderate COVID-19 who are at high risk for progression to severe COVID-19. Veklury was the first drug to get full FDA approval for COVID-19. In July, the FDA approved another supplemental NDA that allowed it to be used across all stages of renal disease.

The FDA based its expansion for all stages of liver disease on the data from a Phase I trial that showed no new safety signals. Hepatic laboratory testing is recommended for all patients and the drug may need to be discontinued if alanine transaminase levels hit 10 times the high end of the normal range or liver inflammation develops.

Aug 21:

Pfizer’s respiratory syncytial virus vaccine Abrysvo won FDA approval for use in pregnant women to prevent RSV-associated lower respiratory tract disease in infants. Abrysvo is an unadjuvanted bivalent RSV prefusion F vaccine composed of two proteins that were selected specifically to optimize against the RSV A and B strains.

The vaccine was first approved in June 2023 for use in adults aged 60 years and above. However, the shot can now also be administered to pregnant women at 32 through 36 weeks of gestation to elicit immunity in their infants.

Data from the Phase III MATISSE trial supported the FDA’s approval. At the study’s prespecified interim analysis, Abrysvo had a vaccine efficacy of 81.8% for preventing medically attended severe RSV-associated lower respiratory tract disease (LRTD) 90 days after birth. This waned slightly to 69.4% at 180 days. The findings were published in The New England Journal of Medicine in April 2023.

Aug 18:

Neurocrine Biosciences got FDA approval to expand Ingrezza’s (valbenazine) label to include chorea in Huntington’s disease. Approved in 2017 to treat tardive dyskinesia, Ingrezza is a selective vesicular monoamine transporter 2 inhibitor.

In the company’s supplemental NDA, Neurocrine included data from the Phase III KINECT-HD trial and the ongoing KINECT-HD2 open-label study to establish the safety and efficacy of Ingrezza in the proposed indication. Data in a Lancet Neurology publication in May showed that Ingrezza treatment significantly reduced scores in the Unified Huntington’s Disease Rating Scale Total Maximal Chorea score by 3.2 units versus placebo, an effect that was statistically significant.

Ingrezza also outperformed placebo on secondary endpoints, including the Clinical Global Impression of Change and Patient Global Impression of Change response status. In clinical studies of Huntington’s disease, treatment-emergent adverse events included somnolence and sedation, urticaria, rash and insomnia.

Aug 18:

Regeneron Pharmaceuticals’ Veopoz (pozelimab) secured FDA approval as the first and only treatment indicated specifically for CHAPLE disease, also known as CD55-deficient protein-losing enteropathy, an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms. A fully human monoclonal antibody, Veopoz is approved for the treatment of adult and pediatric CHAPLE patients 1 year of age and older.

In February 2023, the FDA gave Priority Review to Regeneron’s investigational antibody, which works by binding to the C5 complement factor, thereby disrupting the complement cascade and preventing associated diseases. Regeneron presented Phase II/III data in its application for regulatory approval showing “rapid and sustained normalization” of albumin, one of the key markers of CHAPLE disease, in all 10 patients at 24 weeks. The treatment also eased symptoms such as increased bowel movements and abdominal pain.

Aug 18:

The FDA has expanded the label of Regeneron’s blockbuster eye therapy Eylea (aflibercept), allowing the administration of a higher 8-mg dose. Under the new high-dose regimen, Eylea injections will be given every four weeks for the first three months across all indications.

In diabetic retinopathy, the treatment can be administered every eight to 12 weeks thereafter, while the dosing interval can stretch up to 16 weeks in patients with wet age-related macular degenerationand diabetic macular edema. Eylea was previously approved in these indications but was limited to 2-mg doses.

Aug 16:

After two prior setbacks, Ipsen’s palovarotene finally earned FDA approval to treat fibrodysplasia ossificans progressive (FOP), the first treatment for the ultra-rare bone disease. Now to be sold under the brand name Sohonos, the oral medication is indicated for the reduction of heterotrophic ossification in adults and children with FOP.

The FDA approval is the culmination of a long and bumpy road for Ipsen’s FOP drug. The biotech first tried for an approval in 2021 but voluntarily withdrew its application in August of that year after the regulator asked for more data. Ipsen filed a resubmission a few months later, which the FDA rejected in December 2022 and requested additional information regarding the data that the company had already provided.

The FDA’s approval this week was backed by data from the Phase III MOVE trial, which according to Ipsen is the first and largest multicenter, open-label study in this space involving both adult and pediatric patients. In the trial, palovarotene reduced the annualized heterotrophic ossification by 54% compared with standard of care without introducing new safety signals.

Aug 14:

Following a nearly decade-long effort, Delcath Systems finally won the FDA’s greenlight for its Hepzato Kit for the liver-directed treatment of adult patients with metastatic uveal melanoma. Hepzato is a drug-device combination of melphalan—a well-established chemotherapeutic agent—and the company’s Hepatic Delivery System, which can directly administer the drug to the liver.

Delcath first sought FDA approval in August 2012. More than a year later, in September 2013, the regulator rejected the application, asking the company to perform another well-controlled randomized trial to better determine the safety and efficacy of the investigational product. In February 2023, nearly 10 years later, Delcath resubmitted its NDA.

The application contained data from the Phase III FOCUS study, a single-arm, open-label trial which enrolled 91 patients who were treated every six to eight weeks for up to a maximum of six cycles. FOCUS found that the drug-device combination had an overall response rate of 36.3%, including seven complete responders and 26 partial responders, with treatment response lasting for a median of 14 months. Patients treated with Hepzato saw a 73.6% disease control rate.

Aug 14:

In what is expected to become a multibillion-dollar blockbuster, Pfizer’s BCMAxCD3 bispecific antibody Elrexfio (elranatamab-bcmm) has secured an FDA accelerated approval as another off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy. Elrexfio is being touted by Pfizer as the first off-the-shelf, ready-to-use fixed-dose subcutaneous therapeutic that targets the BCMA protein.

The FDA’s accelerated approval was supported by data from the Phase II MagnetisMM-3 study, which found that in heavily pretreated RRMM patients who had not yet received BCMA-directed therapy, Elrexfio had an overall response rate of 58%, with around 82% of responders maintaining improvements for at least nine months. In those with prior exposures to BCMA-directed therapies, Elrexfio elicited an overall response rate of 33%. The therapy comes with a boxed warning for cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.

Aug 14:

Biotech company Revance Therapeutics has received its first therapeutic indication for Daxxify (daxibotulinumtoxinA-lanm) for the treatment of cervical dystonia in adults. Revance won approval based on data from the Phase III ASPEN 1 and ASPEN OLS trials in which Daxxify was found to be safe and effective at two separate injected doses with a median duration of effect of 24 and 20.3 weeks for the respective groups.

Daxxify, a neuromuscular blocking agent and acetylcholine release inhibitor that’s positioned as a rival to AbbVie’s Botox, was approved in September 2022 for the temporary improvement of moderate to severe frown lines in adults. According to Revance, the total U.S. therapeutic neuromodulator market opportunity for Daxxify is $2.5 billion, including the $345 million cervical dystonia market.

Aug 11:

J&J’s Janssen got the FDA’s greenlight for its PARP inhibitor Akeega (niraparib and abiraterone acetate), which is now authorized to treat BRCA-mutated metastatic castration-resistant prostate cancer. Akeega is the first dual-action tablet that combines the activity of a PARP inhibitor with abiraterone acetate, an androgen biosynthesis inhibitor sold by the company under the brand name Zytiga.

The approval covers a combination regimen of Akeega with prednisone and is based on data from the Phase III MAGNITUDE study, a randomized, double-blinded and placebo-controlled trial with 765 participants. Compared with Zytiga plus prednisone, the Akeega-based regimen significantly improved radiographic progression-free survival by 47% in BRCA-positive patients.

Aug 10:

Janssen also won the FDA’s accelerated approval for its first-in-class bispecific T cell engager Talvey (talquetamab) as a treatment for relapsed or refractory multiple myeloma. By targeting both the CD3 and the GPRC5D proteins, Talvey works by bringing together T cells and myeloma cells, allowing the body’s immune system to exert its anti-cancer effects.

The accelerated approval was based on a Phase II study in which an overall response rate of 73.6% was seen in patients with at least four prior lines of therapy. Response was durable for a median of 9.5 months in the lower dosing group. Median duration of response was not yet reached in the higher dose arm. Like Pfizer’s Elrexfio, Talvey comes with a boxed warning for cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.

Aug 9:

Nearly three years after first winning accelerated FDA approval of Gavreto (pralsetinib) in metastatic non-small cell lung cancer (NSCLC) with RET fusions, Genentech (Roche) and Blueprint Medicines announced the drug’s conversion to full approval.

RET fusions are present in just 1-2% of NSCLC cases, according to Genentech. The original FDA nod was based on an overall response rate (ORR) of 57% and median duration of response (DoR) that had not yet been reached in 114 patients in the Phase I/II ARROW study. The conversion to traditional approval was granted based on an additional 123 patients and 25 months of further follow-up. Treatment-naïve patients saw a median DoR of 13.4 months with a 78% ORR, while patients previously treated with platinum-based chemotherapy had an ORR of 63% and DoR of 38.8 months.

Aug 9:

Shares of Galera Therapeutics plummeted more than 80% Wednesday after the FDA rejected the company’s application for avasopasem manganese (avasopasem). The drug was being proposed to treat severe oral mucositis (SOM)—or mouth sores—resulting from radiotherapy in patients with head and neck cancer.

In its Complete Response Letter, FDA said results from the Phase III ROMAN trial, in combination with the GT-201 trial, were “not sufficiently persuasive to establish substantial evidence of avasopasem’s effectiveness and safety” in reducing these mouth sores. Galera will need to provide results from an additional clinical trial in order to resubmit the application, the Malvern, Penn–based company said in a press release. Impacts for the company’s workforce were swift, as Galera announced it would reduce its numbers by approximately 70% in order to extend its cash runway.

Aug 4:

In another milestone moment, the FDA approved Biogen and Sage Therapeutics’ zuranolone—henceforth Zurzuvae—as the first pill for postpartum depression (PPD). Zurzuvae is only the second treatment for this indication and the first pill that can be taken at home. The drug—a neuroactive steroid that works as a positive allosteric modulator of GABA-A receptors—also acts much more quickly than other approved depression treatments, with improved symptoms seen in trials in as few as three days.

Despite the significance of the approval, Sage and Biogen had sought a much larger slice of the depression market, but this will have to wait as the FDA rejected zuranolone for the treatment of major depressive disorder (MDD). In its Complete Response Letter, the FDA stated that the application did not provide “substantial evidence of effectiveness” to support its approval and that further research would be required. On a conference call following the decision, Sage CEO Barry Greene said only that the companies are “reviewing the feedback and evaluating next steps.”

Aug 4:

Mesoblast will have to provide more data before the FDA may approve its BLA for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). In response to the FDA’s complete response letter (CRL), announced Friday by Mesoblast, the New York–based biopharma will “conduct a targeted, controlled study in the highest-risk adults with the greatest mortality.” Mesoblast noted that the adult study is in line with its overall commercial strategy, which involves a progression from pediatric to adult SR-aGVHD indications. This is the second rejection for remestemcel-L, after the FDA first turned down Mesoblast’s application in 2020, despite a 9-1 advisory committee vote in its favor.

Aug 3:

In 2019, the FDA approved the first vaccine for Ebola, a deadly viral hemorrhagic fever, for adults 18 years and older. Now, that vaccine—Ervebo, developed by Merck—is available to children 12 months and older. The FDA’s decision follows a recommendation on July 20 by the European Medicines Agency’s Committee for Medicinal Products for Human Use to expand Ervebo’s label to children one year and older. The vaccine is only FDA-approved to protect against the Zaire ebolavirus and not other species of Ebola or Marburgvirus and the duration of protection is unknown, according to Merck.

Aug 2:

Taiho Oncology picked up a label expansion for its cancer drug Lonsurf (tipiracil), alone or as part of a combination regimen with Roche’s Avastin (bevacizumab) in metastatic colorectal cancer (mCRC). The nod came nearly eight years after Lonsurf first won approval as a monotherapy for mCRC in Sept. 2015. The expansion pertains to patients who had previously been treated with an anti-VEGF biologic agent and fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. Patients whose cancer has the wild-type RAS protein and who have previously received an anti-EGFR therapy are also eligible for the new combination.

The FDA’s decision was supported by the Phase III SUNLIGHT trial, where the Lonsurf–Avastin combination led to a median overall survival of 10.8 months versus 7.5 months on Lonsurf monotherapy, for a 39% reduction in risk of death.

July 31:

GSK’s Jemperli (dostarlimab-gxly) won FDA approval as a frontline treatment for primary advanced or recurrent endometrial cancer. The regulatory nod is the first new frontline treatment for patients whose cancer is mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) in decades, according to GSK. Jemperli is also the first immuno-oncology treatment and PD-1 inhibitor to be authorized for frontline use in this patient population.

Jemperli, an anti-PD-1 antibody, was first approved in April 2021 for recurrent or advanced dMMR endometrial cancer. The FDA accepted the supplemental BLA to move the drug into the frontline setting on June 6 and set a target action date of Sept. 23, meaning Monday’s approval came nearly two months ahead of schedule.

July 29:

Citius Pharmaceuticals’ plans to bring a reformulated version of Eisai’s withdrawn cancer drug Ontak were put on hold on Saturday after the FDA rejected its BLA for Lymphir (denileukin diftitox). Citius acquired the drug from Dr. Reddy’s Laboratories in Sept. 2021.

An engineered IL-2-diphtheria toxin fusion protein being developed to treat relapsed or refractory cutaneous T-cell lymphoma (CTCL), Lymphir specifically binds to IL-2 receptors to precisely deliver its toxic payload, thereby preventing protein synthesis in malignant T cells. The drug also targets the immunosuppressive regulatory T cells, which allows the body to mount a stronger immune response against the cancer.

In its announcement of the Complete Response Letter, Citius said the FDA will require it to “incorporate enhanced product testing and additional controls” into the BLA. No clinical efficacy or safety issues were raised and Citius stated it will continue working toward FDA approval of Lymphir.

July 28:

The FDA approved RiVive, a 3 milligram (mg) naloxone hydrochloride nasal spray manufactured by Harm Reduction Therapeutics, as the second over-the-counter (OTC) naloxone rescue option for opioid overdose. The approval comes just four months after the regulator greenlit Narcan, a 4-mg naloxone hydrochloride nasal spray, as the first OTC naloxone product. Narcan is manufactured by Emergent BioSolutions. Naloxone rapidly reverses the effects of opioid overdose—a long-running epidemic that claimed the lives of more than 105,000 people in the U.S. in the year ending in February 2023, according to the FDA.

In a press release announcing RiVive’s approval, FDA Commissioner Robert Califf stated the regulator’s commitment to making naloxone accessible and encouraged other manufacturers of these products to discuss potential nonprescription development programs with the FDA.

July 26:

Octapharma secured approval for Balfaxar (prothrombin complex concentrate) for the reversal of the blood-thinner Warfarin in emergency surgery and invasive procedures. Warfarin increases the risk of bleeding—a serious complication during surgery. Balfaxar works to quickly increase blood levels of key clotting factors and antithrombotic proteins. A lyophilized powder, it is reconstituted in a device developed by Octapharma.

While Balfaxar met the primary endpoint of hemostatic efficacy in a Phase III study and was non-inferior to the comparator, Kcentra, fatal and non-fatal arterial and venous thromboembolic complications were observed in clinical trials and post-marketing studies, and healthcare providers are advised to monitor patients for signs of thromboembolic events.

The approval is the third for Balfaxar, which is already marketed in Canada and the EU as octaplex.

July 25:

People with an eyelid disease called demodex blepharitis now have a treatment option as the FDA approved Tarsus Pharmaceuticals’ Xdemvy. A prescription eyedrop, Xdemvy aims to eradicate the root cause of the disease—demodex mites that burrow in the eyelash follicles of sufferers. Xdemvy is the first therapy approved for the condition.

Approval for Xdemvy was granted based on two randomized, multicenter, double-masked, vehicle-controlled studies of 833 patients, in which 415 patients received the treatment. A significant improvement was seen in each study by day 43, Tarsus stated in its approval announcement, adding that the therapy was “generally safe and well-tolerated.”

Tarsus will have a significant market for Xdemvy as demodex blepharitis affects around 25 million people in the U.S. and accounts for more than two-thirds of all blepharitis cases, according to Tarsus.

July 24:

The FDA has approved the first treatment for adults and children two years and older with molluscum contagiosum, a viral skin infection that affects around 6 million people in the U.S. every year. A topical solution developed by Verrica Pharmaceuticals, YCANTH is a drug-device combination that contains a GMP-controlled formulation of cantharidin, a substance derived from the blister beetle Cantharis vesicatoria. It works by causing a blister to form on the wart or growth, eventually lifting it off the skin.

YCANTH’s approval is based on data from two randomized, double-blind, multicenter Phase III trials. In each trial, a clinically and statistically significant number of patients treated with YCANTH achieved complete clearance of all treatable molluscum lesions, meeting the primary endpoint. No serious adverse reactions were reported in either trial. The company expects to make YCANTH available to patients and caregivers by September, Ted White, Verrica’s president and CEO, said in a statement.

July 20:

Four years after it was rejected by the FDA due to safety concerns, Daiichi Sankyo’s Vanflyta (quizartinib) won approval to treat patients with acute myeloid leukemia that has a FLT3-ITD gene mutation. In 2019, an FDA advisory committee voted that the benefit conferred by quizartinib did not outweigh the safety risks. Thursday’s approval comes with a Boxed Warning for three heart disorders: QT prolongation, torsades de pointes and cardiac arrest, and will only be available through a restricted Risk Evaluation and Mitigation Strategy (REMS) program.

Daiichi Sankyo supported its NDA, accepted last Fall, with data from a Phase III trial that showed the quizartinib regimen—combined with standard induction and consolidation chemotherapy and continued as a single agent)—reduced the risk of death by 22.4% compared to patients on chemotherapy alone. After 39.2 months of follow-up, quizartinib more than doubled the overall survival advantage, the company stated in a press release.

July 20:

Emergent BioSolutions secured approval for Cyfendus, a vaccine intended for use after exposure to bacillus anthracis, a bacterium that causes anthrax. Cyfendus is required to be used in combination with “recommended” antibacterial drugs, according to Emergent’s announcement. The approval was based on a series of studies, including a pivotal Phase III trial assessing the vaccine’s consistency, immunogenicity and safety in healthy adults. Even before the approval, Emergent had been supplying Cyfendus to the U.S. Department of Health and Human Services under pre-Emergency Use Authorization for four years.

July 17:

Infants and children at severe risk of RSV have another treatment option after the FDA approved Sanofi and AstraZeneca’s Beyfortus (nirsevimab). A monoclonal antibody, Beyfortus is just the second RSV drug authorized in the U.S. for high-risk children, the other being Synagis (palivizumab), which was approved in 1998. The companies’ bid for approval was backed by three late-stage trials that demonstrated the antibody’s safety and efficacy. Beyfortus will be available in the U.S. ahead of the 2023/2024 RSV season for children entering their first RSV season and those at severe risk up to 24 months, Sanofi stated in a press release.

July 13:

The FDA approved Perrigo Company’s Opill (norgestrel)—a progestin-only daily oral contraceptive—as the first-ever birth control pill available over the counter in the U.S. Perrigo expects Opill to be available online and in-person at drug stores, convenience stores and grocery stores in the first quarter of 2024. In a prepared statement, Perrigo President and CEO Patrick Lockwood-Taylor said the approval “marks a truly momentous day for women’s health nationwide.”

July 6:

A short workweek in the U.S. ended with big news as the FDA granted traditional approval to the first anti-amyloid antibody—and the first disease-altering drug—for Alzheimer’s disease. The accelerated approval for Eisai and Biogen’s Leqembi (lecanemab) was converted to a full approval based on results from the confirmatory Clarity-AD trial, which the FDA said in its statement verified the drug’s benefit. With the approval, Medicare coverage for Leqembi is expected to begin right away, with the requirement of a patient registry intended to collect further information on the effectiveness of this drug class in Alzheimer’s.

July 3:

Amneal Pharmaceuticals received a Complete Response Letter for IPX203—a novel oral formulation of carbidopa/levodopa (CD/LD), a well-established combination for the management of Parkinson’s disease. In its rejection letter, the FDA said that while Amneal was able to adequately establish the safety of levodopa, it was not able to sufficiently do so for carbidopa. The regulator has requested additional pharmacokinetic data. The New Jersey–based company stated it plans to meet with the FDA to determine the best path forward for the treatment.

June 29:

Capping a busy week, the FDA approved BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) as the first gene therapy for adults with severe hemophilia A. Hemophilia is a rare genetic bleeding disorder caused by a mutation in the gene that encodes factor VIII (FVIII), which is necessary for blood to clot. A one-time gene therapy, Roctavian contains a healthy gene for factor VIII. Delivered through an adeno-associated virus (AAV) vector, the gene is expressed in the liver to increase blood levels of FVIII, thereby reducing the risk of uncontrolled bleeding.

June 28:

The FDA notched another milestone with the approval of the first cellular therapy for type 1 diabetes. Lantidra (donislecel) developed by Chicago-based CellTrans, is a cell therapy made from the pancreatic islet cells of deceased donors. It is intended for adult patients whose repeated hypoglycemic episodes leave them unable to hit average blood glucose levels. In a clinical trial of 30 patients, 21 were insulin-free for at least a year; 11 didn’t require insulin for between one and five years and 10 were insulin-free for more than five years. Five patients failed to achieve any days of insulin independence.

June 28:

Pfizer’s bet on OPKO Health’s human growth hormone analog paid off as the FDA approved the treatment—which will be marketed as Ngenla—to treat children whose production of growth hormones is impaired. Pfizer purchased exclusive global commercialization rights for the then-experimental treatment in December 2014 for $295 million upfront and a promise of up to $275 million in milestones. This was the partners’ second try for FDA approval after the regulator rejected Ngenla’s first bid in January 2022. Pfizer and OPKO did not state the reasons for the initial denial.

June 28:

The FDA declined to approve Eton Pharmaceuticals’dehydrated alcohol injection for the treatment of methanol poisoning, citing concerns related “primarily to Chemistry Manufacturing and Controls.” This is the second Complete Response Letter issued to Eton for this proposed treatment. In the first, issued in March 2021, the FDA indicated that travel restrictions due to the COVID-19 pandemic prevented a timely inspection of the company’s European contract manufacturing site.

June 27:

The FDA approved UCB’s Rystiggo (rozanolixizumab), a subcutaneously administered humanized IgG4 monoclonal antibody, to treat generalized myasthenia gravis, a rare muscle-wasting autoimmune disease. Rystiggo is the only treatment approved to treat patients who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive. The regulatory green light was based on a Phase III trial that showed Rystiggo led to significant improvements in symptoms related to breathing, talking, swallowing and rising from a chair.

June 23:

The FDA greenlit Pfizer’s Litfulo (ritlecitinib) for the treatment of patients as young as 12 years with severe alopecia areata, an autoimmune disease characterized by patchy or complete hair loss on the scalp, face or body. Litfulo inhibits Janus kinase 3 and the tyrosine kinase and is believed to work by blocking the signaling of cytokines and cytolytic activity of T cells, which are implicated in alopecia areata.

June 22:

In one of the year’s most-anticipated regulatory decisions, the FDA approved Sarepta’s Elevidys—formerly SRP-9001—for children 4 to 5 years with Duchenne muscular dystrophy. Elevidys is the first-ever gene therapy for DMD, a neuromuscular disease characterized by progressive muscle weakness and atrophy that strikes primarily young boys. The gene therapy was approved via the FDA’s accelerated approval pathway based on data that established it increased the expression of the Elevidys micro-dystrophin protein.

June 21:

Eli Lilly and Boehringer Ingleheim’s Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride) picked up another indication, becoming the first and only SGLT2 inhibitors approved for children 10 years and older with type 2 diabetes. Previously, there had only been one oral drug for this indication: metformin, which was approved in 2000.

June 20:

Argenx will bring to market the first subcutaneous injectable for generalized myasthenia gravis (gMG), a rare muscle-wasting autoimmune disease. Vyvgart (efgartigimod) was first approved in 2021 as a one-hour intravenous infusion. Vyvgart is an antibody fragment that binds to the neonatal Fc receptor to prevent recycling of immunoglobulin G back into the blood. This includes a reduction in abnormal AChR antibodies, which are present in approximately 85% of gMG patients. AChR antibodies block the acetylcholine receptors from being able to receive signals from the nerve to stimulate a muscular response. Their normalization should ostensibly improve muscular function.

Heather McKenzie is a senior editor at BioSpace, focusing on neuroscience, oncology and gene therapy. You can reach her at heather.mckenzie@biospace.com. Follow her on LinkedIn and Twitter @chicat08.