uniQure BLA for Huntington's gene therapy accepted by FDA; target action date May 1, 2027
uniQure N.V. said the FDA accepted its Biologics License Application for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease, and granted Priority Review with a PDUFA target action date of May 1, 2027. The FDA also said it is tentatively planning an Advisory Committee meeting, with dates to be set later, and the company and FDA have aligned on the design of a confirmatory trial.
Key points
- FDA accepted the BLA under the Accelerated Approval pathway and granted Priority Review
- PDUFA target action date is May 1, 2027
- FDA tentatively plans to discuss the application at an Advisory Committee meeting; the company will be notified once the date is set
- Confirmatory trial design: randomized, standard-of-care, about 200 patients in a 2:1 ratio, Total Functional Capacity at 36 months as primary endpoint; U.S. patient screening planned to start by end of 2026
The filing
Written by AI on Nov 9, 2026, 8:38 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on November 02, 2026.
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