FDA accepts uniQure's Huntington's gene therapy BLA, grants Priority Review with PDUFA date May 1, 2027

uniQure N.V.

• FDA accepted for review the BLA for ifezuntirgene inilparvovec (AMT-130) under the Accelerated Approval pathway with Priority Review; PDUFA target action date May 1, 2027.
• In its acceptance letter, FDA said it is tentatively planning to discuss the application at an Advisory Committee meeting; date and timelines not yet finalized.
• Confirmatory study aligned with FDA: ~200 patients randomized 2:1 to ifezuntirgene inilparvovec vs standard of care, Total Functional Capacity primary endpoint at 36 months.
• BLA rests on 36-month Phase I/II data vs a propensity score-matched external control from the Enroll-HD database, disclosed Sept 24, 2025; 51 patients dosed in the program.
• First Huntington's therapy with Breakthrough Therapy and RMAT designations; also holds Fast Track. US confirmatory-trial screening planned by end of 2026.