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.
FEATURED STORIES
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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 issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
After receiving a letter directly from CBER Director Vinay Prasad, Moderna said the FDA had previously signed off on the use of a licensed flu vaccine as a comparator for a Phase 3 study of mRNA-1010.
The agency flagged several violations at a compounding pharmacy owned by Hims & Hers, including “infestation by rodents, birds insects, and other vermin.”
The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.
The FDA underwent significant changes during the first year of the second Trump administration, directly affecting business risk and opportunity. Understanding key 2025 trends will be critical to developing regulatory strategies and maximizing opportunities for success.
Regulatory challenges have been even more top of mind than usual given recent upheaval at the FDA. BioSpace spoke to three industry experts about key issues, which include applying new artificial intelligence guidance. The experts also shared advice for working with regulators.
After review, Amgen is certain that Tavneos is effective and has a favorable benefit-risk profile. The company informed the FDA on January 28 that they would not pull the drug.
Rep. Jake Auchincloss of Massachusetts said the Commissioner’s National Priority Voucher program did not receive congressional backing. The FDA has also not yet made disclosures for eight senior reviewers, according to Auchincloss.
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, while ending a three-day partial government shutdown.