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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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 FDA previously rejected Zycubo for Menkes disease in October last year, citing issues with the drug’s manufacturing facility.
Drugmakers told the FDA that inflexible post-approval change requirements are among the top regulatory barriers to the reshoring of pharmaceutical manufacturing.
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
After greenlighting 56 novel therapeutics in 2025, four notable applications continue to await the agency’s action after being delayed from the fourth quarter last year.
The DC-based biopharma disputed the FDA’s conclusions regarding the data provided in its supplemental application for Hetlioz and promised to keep pushing for an approval.
Last month, the FDA declined to approve Sanofi’s tolebrutinib for a specific form of multiple sclerosis. In a recently published complete response letter, the agency detailed its reasoning behind the rejection.
In a year that saw advisory committees placed under a particularly bright microscope at the FDA, the agency held fewer meetings than usual and agreed with its advisors only 57% of the time, Jefferies reported.
After a tumultuous year, experts call for stability while anticipating the first fruits of policies intended to expedite approvals for rare disease drugs.
The FDA’s announcement that it will phase out in vivo testing requirements for monoclonal antibodies marks a seismic shift. Here’s how industry can adapt.
Jefferies analysts envision a steady launch curve that could ultimately drive meaningful sales from people who are dissatisfied with existing treatments.