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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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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Both companies received agency requests for more evidence of the effectiveness of their therapies.
Analysts called the approval a much-needed win for Novo Nordisk, but warned that the company could struggle to grow sales once rival drugs come to market.
The biologics center director reportedly became personally involved after the team reviewing the rare blood disorder filing asked for an extension to the CNPV-accelerated timeline.
Stifel analysts said the label for cardiac myosin inhibitor Myqorzo is in line with their expectations and is differentiated compared with BMS’ Camzyos.
Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry.
The filing comes as Novo fights tooth-and-nail with rival Lilly to regain its footing at the top of the weight loss market.
The tickets could go to the lipid-lowering pill enlicitide decanoate and the antibody-drug conjugate sacituzumab tirumotecan, though a spokesperson for the HHS did not confirm the news.
The FDA rejected the use of Exdensur, however, for chronic rhinosinusitis with nasal polyps.
Speaking on Bloomberg TV on Monday, FDA Commissioner Marty Makary contradicted recent reporting that suggested the agency was planning to add a black box label—its most serious warning—to COVID-19 vaccines.
Analysts at Jefferies called the approval “highly significant,” estimating it could add $2 billion to $3 billion to peak Enhertu sales.