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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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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The disagreement between Moderna and the FDA has reached a resolution just eight days after the biotech received a Refusal-to-File letter in response to its application for mRNA-1010. Moderna will now seek approval of the vaccine based on age.
Regulatory uncertainty is no longer background noise. It is a material investment risk that reshapes how capital is deployed and pipelines are prioritized.
Disc Medicine’s leadership tried to express optimism that its rare disease therapy bitopertin can be approved based on a Phase 3 trial set to begin shortly. However, analysts are worried that the protocol was developed with former FDA leaders.
Johns Hopkins’ Thomas Hartung discusses how drug discovery and development will change under evolving regulatory policies that embrace AI technology as well as organoid and other non-animal models of human biology.
The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is “part of a disturbing pattern” of moving regulatory goalposts, according to Clay Alspach, executive director of the Alliance for mRNA Medicines. Meanwhile, streamlined communications with regulators in other countries pave the way for rapid uptake of novel modalities.
The FDA in October 2025 granted bitopertin its Commissioner’s National Priority Voucher but after a shortened review time has decided the data did not support regulatory approval for treating erythropoietic protoporphyria.
Richard Pazdur had spent 26 years at the FDA before stepping down last year due to disagreements with Commissioner Marty Makary about lowering the number of clinical trials required for a new drug application, Pazdur told the media.
Through its AI Consortium and global forums, DIA is translating high‑level AI guidance into concrete workflows that match oversight to risk, from low‑stakes automation to decision‑critical regulatory analyses.
The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
Vinay Prasad, the FDA’s Center for Biologics Evaluation and Research head, is accused of interpersonal impropriety as pushback builds against his decision to reject Moderna’s influenza vaccine candidate.