FDA
After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application.
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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.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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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.
FDA veteran Peter Marks will now shape the future of Eli Lilly’s vaccines work after the buys of Curevo, LimmaTech Biologics and Vaccine Company for up to $3.8 billion total.