FDA
UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?
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The FDA has expanded the regulatory toolkit during President Donald Trump’s second term, adding new mechanisms for rare diseases while putting the Biden-era platform technology designation into action.
As the FDA tries to clarify its intent for former FDA Commissioner Marty Makary’s plausible mechanism framework for bespoke therapies, experts emphasize the importance of expanding its scope to encompass rare diseases that affect more than just one or a few individuals.
The FDA’s decision last year to make complete response letters public provides new insight into why therapies sometimes fail to get the regulatory greenlight. Analysts say the information could help sponsors refine their regulatory strategies.
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The FDA rejected bitopertin in February amid reports of skepticism from former CBER director Vinay Prasad, who has since departed the agency.
The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation.
The FDA hasn’t been transparent and open enough with how it has implemented the Commissioner’s National Priority Voucher program, patient, industry and trade groups said Thursday.
FDA inspectors found that Medline, which raised billions of dollars last year, failed to prevent repeat bacterial contamination of finished drug products.
A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.
FDA inspectors found Alchymars failed to adequately maintain equipment used to make active pharmaceutical ingredients for the U.S. market. Over-the-counter drugmakers GC America and Sato Pharmaceutical also received warning letters.
Replimune’s resubmission for RP1 for melanoma comes amid the departures of FDA leaders in place at the time of the drug’s first two rejections, last summer and again in April.
Beren Therapeutics is seeking approval in Neimann-Pick disease type C, an application the FDA was set to decide on by August 17. The new target action date is November 17.
Decnupaz is the first antibody-drug conjugate for blastic plasmacytoid dendritic cell neoplasm, an ultra-rare and aggressive blood cancer.
The FDA’s priority review acceptance of BridgeBio’s BBP-418 is another step toward what William Blair previously dubbed a “diversified commercial portfolio.” It also adds to the rapidly building momentum in muscular dystrophy more broadly.