Regulatory
Facing increasing pressure from both industry and the White House, FDA Commissioner Marty Makary said the strong bad press against him is “corporate spin” and that the agency has “followed the science.”
First quarter earnings continue to arrive, with analysts demanding more from cautious Pfizer and Eli Lilly expecting more revenue; the FDA taps Katherine Szarama as Vinay Prasad’s controversial FDA tenure ends; oncology veterans miss Richard Pazdur at the agency’s first adcomm in nine months; and QurAlis and Corcept Therapeutics spark renewed hope in ALS.
The FDA is reportedly down to a handful of final candidates to lead CBER, with a potential selection expected in the coming month or two. For now, the appointment of acting director Katherine Szarama has not allayed the industry’s concerns.
Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than early-stage clinical trials of candidates from REGENXBIO, Excision BioTherapeutics and Intellia Therapeutics.
The advisory committee meeting—the FDA’s first drug-related adcomm in nine months—could have been a “more conceptual discussion” about the design of AstraZeneca’s Phase 3 trial of camizestrant in HER2-negative advanced breast cancer, former cancer regulator Harpreet Singh told BioSpace.
Veppanu, the first PROTAC therapy approved by the FDA, improved progression free survival by 43% versus AstraZeneca’s Faslodex but showed no such significant benefit in the intention-to-treat analysis.
The FDA is looking at a slew of label expansions this month, including one that could open up home-based treatments for Alzheimer’s disease.
Members of the FDA’s Oncologic Drugs Advisory Committee questioned the design of AstraZeneca’s Phase 3 trial of camizestrant, which involved switching treatments at the point of mutation detection, as opposed to the current practice of changing regimens upon disease progression.
UniQure plans to submit AMT-130 to the U.K.’s Medicines and Healthcare products Regulatory Agency in the third quarter of 2026 based on Phase 1/2 data showing a 75% slowing of disease—the same data the FDA has deemed unacceptable for a biologics license application.
The patient death occurred outside the U.S. and was deemed unrelated to Newron Pharmaceuticals’ investigational schizophrenia drug.
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