FDA
Aiming to protect patients, the FDA sent lawmakers a wish list of legislative proposals intended to clarify and expand its oversight of updates to approved drug production processes.
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The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.
The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher.
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According to reporting from multiple outlets, Richard Pazdur, head of the Oncology Center of Excellence at CDER, opposed the consensus opinion of CBER staff to approve the drug. Replimune’s stock has dropped precipitously since the rejection.
Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public.
The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome.
As analysts parsed news of Vinay Prasad’s ouster, worries over drug approval delays, cell and gene therapy impacts and more were top of mind.
The FDA’s dramatic summer continues to unfold as news broke late Tuesday evening that Vinay Prasad will depart the agency, where he had been the head of the Center for Biologics Evaluation and Research for less than three months.
The swift FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety study, as had been feared, Jefferies analysts said Monday.
The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics.
The new target action date for Blenrep, which GSK is proposing for the second-line treatment of relapsed or refractory multiple myeloma, is Oct. 23.
While FDA Commissioner Marty Makary emphasizes learning and humility, the FDA has systematically removed the very experience that would make change possible.