FDA
FDA Commissioner Marty Makary last week announced a directive that would limit industry participation in the agency’s advisory committees. But not only do company reps serve only as non-voting members, a 1997 law actually requires industry involvement.
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As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
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The approval concludes what has been a difficult regulatory path for Ryoncil, which suffered FDA rejections in 2020 and 2023.
The letters come amid the Outsourcing Facilities Association’s ongoing lawsuit against the FDA over the regulator’s decision to end the shortage for tirzepatide.
The FDA cited issues with a manufacturing facility as the reason for the rejection. J&J is currently “working closely” with the regulator to resolve these problems.
The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The company faced the same hurdle for an earlier neuromuscular candidiate in 2023.
The EMA approved a kidney disease–related label expansion for the blockbuster GLP-1 drug after a study showed reduced risk of death by 20%.
The FDA’s year-end rush includes nine target action dates, mostly for rare disease and cancer therapies.
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.
The program has recently become controversial as a number of high-profile advanced approvals were granted only for the drugs to fail a confirmatory trial later on. Now, the FDA has clearly laid out expectations.
The agency’s warning letter outlines Applied Therapeutics’ failure to provide adequate information regarding the clinical trial—including a dosing error for govorestat—claims that the company said it had already addressed.
Novartis, Gilead, Roche and Takeda commit to new partners in a spate of mid-sized collaborations this week. Meanwhile, Applied Therapeutics’ stock tanks 80% after govorestat is denied approval, Intra-Cellular Therapies seeks to expand Caplyta into major depressive disorder and the FDA investigates the safety of bluebird bio’s Skysona.