Kyverna plans to submit mivocabtagene autoleucel to the FDA for approval in the first half of 2026. If approved, it would be the first CAR T therapy for an autoimmune disease.
Analysts at Jefferies called the approval “highly significant,” estimating it could add $2 billion to $3 billion to peak Enhertu sales.
Ambros Therapeutics’ non-opioid bisphosphonate analgesic, already approved in Italy, will soon begin a pivotal test in the U.S.
Johnson & Johnson, which did not apply for the national priority voucher, was granted the ticket based on results from a Phase III study testing Tecvayli plus Darzalex in patients with relapsed or refractory multiple myeloma.
Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s approach to pre-approval inspections.
Varegacestat, a gamma secretase inhibitor, significantly improved progression-free survival while also meeting all key secondary endpoints in the pivotal RINGSIDE trial. Immunome is planning an FDA application for the second quarter of 2026.
FEATURED STORIES
In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former regulator insists that a “failed trial is not a failed drug.”
As clinical research evolves, disruptive trial designs are reshaping how oncology advances. By integrating novel methodologies, strategic innovation, and patient-centered approaches, MEDSIR is driving a new era in cancer research, one that accelerates development, enhances efficiency, and delivers more meaningful therapeutic progress for patients worldwide.
Coming up in the back half of December, the FDA will issue a verdict on Vanda Pharmaceuticals’ gastroparesis drug tradipitant, which it rejected last September, triggering a very public dispute with the company.
Every year in biopharma brings its share of grueling defeats, and 2025 was no different, especially for companies targeting neurological diseases. Some failures split up partners, and one particularly egregious case even led to the demise of an entire company.
With five CDER leaders in one year and regulatory proposals coming “by fiat,” the FDA is only making it more difficult to bring therapies to patients.
LATEST PODCASTS
In this episode of Denatured presented by AnaptysBio, Jennifer Smith-Parker speaks to Dr. Joe Murray, Mayo Clinic; Marilyn Geller, the Celiac Disease Foundation; and Dr. Paul Lizzul, AnaptysBio, about the challenges and opportunities facing celiac disease treatment.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for new approach methologies (NAMs) development.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
DEALS
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Sanofi bought Dren’s DR-0201 program earlier this year for $600 million upfront and is running two Phase I trials in undisclosed inflammatory indications. -
With $6 billion left in firepower, Pfizer is planning transactions in the hundreds of millions to the low-billions range, particularly in internal medicine and immunology and inflammation, Guggenheim reported. -
Pfizer apparently had more in the tank after the high-profile battle to acquire Metsera earlier this fall. The company has licensed a new GLP-1 from YaoPharma. -
What China is accomplishing in R&D “has implications for everyone playing in the R&D or innovation world,” McKinsey’s Fangning Zhang says. -
The centerpiece of the deal is the in vivo editor TSRA-196, which in preclinical studies has shown robust editing at SERPINA1, the locus linked to alpha-1 antitrypsin deficiency.
WEIGHT LOSS
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Analysts were hoping for a safety profile similar to what was achieved in Phase II but an abnormal sense of touch, called dysesthesia, has emerged in the late-stage TRIUMPH-4 trial. -
Also on Thursday, Zealand held its Capital Markets Day in London, outlining the strategy for its weight management franchise in the near-term, including launching five products by 2030. -
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more. -
Structure’s aleniglipron elicited over 11% weight loss in a Phase II trial, sending the biotech’s stock up nearly 103% as markets closed on Monday. -
Although still in Phase I, Wave Life Sciences’ injectable RNA weight loss treatment achieved results that impressed analysts, with 4% fat reduction after three months, beating Novo Nordisk’s semaglutide at a similar time point.
POLICY
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The ACIP voted 8-3 to recommend delaying the hepatitis B vaccine, commonly given just after birth. The CDC itself has said the shot is safe and effective. -
Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials. -
In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for new approach methologies (NAMs) development. -
This week’s meeting of the Advisory Committee on Immunization Practices will be led by Kirk Milhoan, a physician and pastor who recently claimed that COVID-19 vaccines contained a contamination that causes cancer.
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U.K.-based pharmas will not face tariffs as long as Donald Trump is president, according to the agreement.
What should you do when belief in the mission remains, but the career path doesn’t?
Looking for a biopharma job? Check out the BioSpace list of 12 top companies hiring life sciences professionals like you.
Employees rarely leave companies for one reason alone. In this column, Kaye/Bassman’s Michael Pietrack shares a framework that helps leaders identify when their team members are thinking about heading for the exit—and how to address it.
Biopharma professionals aren’t typically hired right away, based on a BioSpace LinkedIn poll. In the past year, only about one-third of respondents found employment in three months or less. Several who did share their keys to success.
In a volatile industry, staying put might seem like a smart bet, but job hugging can quietly erode your visibility, growth and future opportunities.
Transparency doesn’t drive people away. It attracts the right ones and keeps them committed. Leadership coach Angela Justice discusses the problem with leaders only selling the upside and the value of setting accurate expectations from the start.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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While new late-stage data point to some liver toxicity signals, analysts at BMO Capital Markets said Tukysa’s efficacy outcomes “appear to more than make up for any safety concerns.” -
While overall survival remains immature, results so far show a clear trend in favor of Roche’s giredestrant. -
TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying. -
The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases. -
GSK and Ideaya first linked up in 2020 to advance novel therapies for solid tumors. It is unclear why the pharma terminated the partnership.
NEUROSCIENCE
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Sanofi’s multiple sclerosis hopeful tolebrutinib faced dual setbacks on Monday, with a late-stage failure in one form of the disease and yet another regulatory setback in another. -
In a first for the Parkinson’s field, AC Immune’s immunotherapy has stabilized key biomarkers that suggest an effect on the disease’s course. -
Analysts at Jefferies see blockbuster potential in zorevunersen in Dravet syndrome, with sales potentially reaching $1 billion to $4 billion. -
The R&D pipeline for depression therapies faced a demoralizing 2025 as five high-profile candidates, including KOR antagonists by Johnson & Johnson and Neumora Therapeutics, flunked late-stage clinical trials, underscoring the persistent challenges of CNS drug development. -
Innovative outcome measures coupled with a focus on patient-centered clinical differentiation can help the biopharma industry make meaningful progress in the highly complex area of neuroscience.
CELL AND GENE THERAPY
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Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval. -
For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products. -
As big pharmas including Takeda and Novo Nordisk flee the cell therapy space and smaller biotechs shutter their operations, these players are sticking around to take the modality as far as it can go. -
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field. -
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
