FDA
As Novo Nordisk cuts 400 jobs at the troubled site, Scholar Rock has seen enough progress that it included the facility in a resubmission for FDA approval.
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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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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Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies.
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking.
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market.
On the FDA’s docket this month are two decisions pushed back from 2025, including one for a rare form of obesity and another for dry eye disease.
This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
FDA Commissioner Marty Makary presented a new idea to staff this week: bonus payments for employees that complete regulatory review processes faster than expected.
Hernexeos is the second drug to secure an FDA approval under the agency’s priority voucher scheme, following in the footsteps of USAntibiotics’ Augmentin XR, which was granted the ticket in December 2025.
Aside from creating a toxic work environment, CBER Director Vinay Prasad has also been accused of berating his staff and retaliating against reviewers who questioned his decisions.
Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients.