FDA Weekly Review looks at the FDA’s actions related to drug approvals, IND approvals, designations and more. Here’s a look at what happened this week.
Sarah Silbiger/Getty Images
BioSpace is excited to introduce FDA Weekly Review, a column that looks at the U.S. Food and Drug Administration’s actions related to drug approvals, IND approvals, Breakthrough and Priority designations and more. Here’s a look at what happened this week.
FDA Approvals
Avadel Pharmaceuticals announced Tuesday that the FDA has granted tentative approval to Lumryz (FT218). This action indicates that the drug met all required quality, safety and efficacy standards, but final approval is pending resolution of a patent dispute with Jazz Pharma. Lumryz is a once-at-bedtime formulation of sodium oxybate for excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy.
The tentative approval was based on data from the Phase III REST-ON trial that showed the drug demonstrated statistically significant and clinically meaningful results in patients with EDS.
Avadel CEO Greg Divis said his company is “pursuing all options to accelerate final approval on or before June 2023 and prepare for commercial launch.”
Eton Pharmaceuticals announced Monday that the FDA has approved Zonisade (zonisamide oral suspension). The drug was included in Eton’s multi-product neurology oral solution partnership with Azurity Pharmaceuticals announced in February 2021. Azurity is responsible for commercializing the drug. Zonisade was approved as adjunctive therapy for partial seizures in adults and pediatric patients 16 years and older with epilepsy.
Amphastar Pharmaceuticals reported FDA approval of its Abbreviated NDA for Vasopressin injection, USP 20 Units/mL, 1mL Single Dose Vial. The drug is indicated to increase blood pressure in adults with vasodilatory shock who remain hypotensive (with low blood pressure) despite fluids and catecholamines. The agency determined vasopressin is bioequivalent and therapeutic equivalent to Par Sterile Products’ Vasostrict.
Pfizer’s Xalkori (crizotinib) was approved for adults and pediatric patients one year and older with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The approval was based on two open-label trials that included 14 pediatric patients from ADVL0912 and seven adults from A8081013. The major efficacy outcome was objective response rate (ORR).
Neuronetics received clearance from the FDA for the use of its transcranial magnetic stimulation (TMS) system, NeuroStar Advanced Therapy for Mental Health, in treating anxiety symptoms in adults with major depressive disorder (MDD), also called anxious depression. The agency accepted Neuronetics’ use of real-world data analyzed from NeuroStar’s TrakStar platform in granting clearance for this indication.
Designations
Celcuity announced that the FDA has granted gedatolisib Breakthrough Therapy designation for the treatment of patients with HR+/HER2- metastatic breast cancer whose disease progressed during treatment with a CDK4/6 therapy and a non-steroidal aromatase inhibitor. The drug is a potential first-in-class pan-PC3KmTOR inhibitor. The breakthrough application was supported by data from a Phase Ib trial assesing the drug’s safety, tolerability and clinical activity in combination with palbociclib and fulvestrant for this indication.
Apellis Pharmaceuticals received Priority Review designation for its new drug application for intravitreal pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration. The PDUFA date is November 26, 2022. The NDA is built on data from the Phase III DERBY and OAKS studies and the Phase II FILLY trial. The drug is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the immune system.
Pliant Therapeutics received Fast Track designation from the FDA for PLN-74809, its oral, dual-selective αvß6 / αvß1 integrin inhibitor, for the potential treatment of primary sclerosing cholangitis (PSC). It is currently being evaluated in the Phase IIa INTEGRIS-PSC clinical trial.
Olema Pharmaceuticals received Fast Track designation from the FDA for OP-1250. The drug is an oral complete estrogen receptor (ER) antagonist and selective ER degrader for the treatment of ER-positive, HER2- metastatic breast cancer that has progressed following one or more lines of endocrine therapy with at least one given in combination with a cyclin-dependent kinase (CDK) 4/6 inhibitor.
New Trials
Alzamend Neuro received a positive written response to its meeting request for a Type B Pre-Investigational New Drug (IND) application with the FDA. The response provides a clear path for the company’s planned clinical development of AL001 for bipolar disorder, major depressive disorder and post-traumatic stress disorder. AL001 is a novel lithium-delivery system, a lithium-salicylate-L-proline engineered ionic cocrystal currently being assessed in a Phase II trial for the oral treatment of patients with dementia resulting from mild, moderate or severe cognitive impairment caused by Alzheimer’s disease.
The MD Anderson Cancer Center and Obsidian Therapeutics received the go-ahead from the FDA for their IND application to conduct a Phase I trial of OBX-115. The drug is an engineered tumor-infiltrating lymphocyte (TIL) therapy being developed as a monotherapy to treat adults with metastatic melanoma who are relapsed or refractory to previous checkpoint therapy treatments.
