FDA
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
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Regulatory uncertainty is no longer background noise. It is a material investment risk that reshapes how capital is deployed and pipelines are prioritized.
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’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.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
The U.S. regulator shared the roadmap for implementing the program, first proposed in August 2025, and teased changes made in response to industry feedback.
The initiative could tackle the first-mover disadvantage some CDMOs believe deters early customers, but leaders at companies including Novo Nordisk see hurdles to implementing the changes.
As biotechs faced investors at the J.P. Morgan Healthcare Conference this week, they emphasized agreement with the FDA on clinical trial design and regulatory pathways to approval. Atara, meanwhile, lamented the agency’s “complete reversal of position” after its therapy for a rare surgical complication was rejected.
Target action dates for drugs sponsored by Sanofi, Boehringer Ingelheim and Disc Medicine have also been pushed back despite assurances of swift reviews under the FDA’s new Commissioner’s National Priority Voucher program.
A more detailed review of data by the FDA showed that GLP-1 drugs do not increase the risk of suicidal ideation or behavior.
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.