FDA
Twelve marketed drugs, including Eli Lilly’s and Novo’s weight loss mega-blockbusters, will require a label change under the new FDA update.
FEATURED STORIES
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
By allowing compounding pharmacies to produce certain unapproved peptides, the Department of Health and Human Services could, in the short term, put patients in harm’s way. Longer term, the policy shift threatens the industry that brings new medicines to market.
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is designed to develop personalized gene therapies more quickly.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
If approved, BridgeBio Pharma’s BBP-418—which, according to one Jefferies analyst, boasts “pretty darn compelling” data—would be the first therapy for limb-girdle muscular dystrophy. The FDA has an action date of Nov. 27.
Under the interim policy, officials do not intend to take action against sites that produce the peptides.
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
Drug developers increasingly have the option of seeking regulatory approval and commercializing a medicine themselves, rather than accepting an early exit offer. Doing so requires a worldwide strategy.
After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aardvark plans to resume testing the candidate next year.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
The approval of Hibsago has created a new market for a handful of biopharmas, including Gilead, Arrowhead, and Vir, to fill in.
The recurrence of violations at multiple sites led the FDA to conclude that Baxter management must improve its oversight and control of manufacturing.