FDA
With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA.
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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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.
Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a need for more rigorous evidence.
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The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay.
According to an internal email, the agency may be in for more consolidation in areas including human resources, communications, travel and acquisitions.
Later this month, HHS Secretary Robert F. Kennedy Jr.'s revamped CDC vaccine advisory committee will discuss RSV vaccination guidelines for the newly approved patient group, high-risk adults 18 through 49. Analysts and other experts have warned that the new panel includes some who have documented anti-vaccine sentiments as well as those who have spoken out against mRNA technology specifically.
Martin Kulldorff and Robert Malone, both outspoken vaccine skeptics, have received compensation for their expert participation in various vaccine-related cases against Merck.
The FDA’s Oncologic Drugs Advisory Committee narrowly voted against the approval of Zusduri, citing the lack of a completely randomized study to back up the application.
Nuvation Bio’s first approved product is Ibtrozi, a CNS-active ROS1 inhibitor that in pivotal studies showed high rates of treatment response in patients with non-small cell lung cancer.
FDA Commissioner Marty Makary and CBER Director Vinay Prasad published an article in JAMA on Tuesday outlining the FDA’s priorities, including accelerating cures and the rapid deployment of artificial intelligence.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
Gilead underscored its faith in the combo therapy and pledged to work with regulators to resolve the hold, which has paused five clinical trials. Gilead also stressed that the hold does not impact any other assets in its HIV pipeline.
Merck’s Enflonsia will go up against Sanofi and AstraZeneca’s Beyfortus, which the partners plan to ship out early in the third quarter.