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FDA accepts BLA for Huntington's gene therapy ifezuntirgene inilparvovec, sets PDUFA date of May 1, 2027
QUREuniQure N.V.
The U.S. Food and Drug Administration has accepted the Biologics License Application for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease, under the Accelerated Approval pathway. The application received Priority Review designation with a Prescription Drug User Fee Act target action date of May 1, 2027. The FDA plans to discuss the application at an Advisory Committee meeting. The BLA is supported by 36-month data from Phase I/II studies compared to external controls. uniQure and the FDA have aligned on a confirmatory study design involving approximately 200 patients, with screening expected to begin in the U.S. by the end of 2026. Ifezuntirgene inilparvovec holds Breakthrough Therapy, RMAT, and Fast Track designations. uniQure is a gene therapy company developing treatments for severe diseases including hemophilia B and Huntington's disease.