AMT-130

November 2, 2026: uniQure N.V. (NASDAQ: QURE) said the FDA accepted for review its BLA for ifezuntirgene inilparvovec (AMT-130) for Huntington’s disease under the Accelerated Approval pathway, granted Priority Review, and set a PDUFA target action date of May 1, 2027. The FDA said it is tentatively planning an Advisory Committee meeting, with timing to be announced.

The BLA is supported by previously announced 36-month Phase I/II data versus a propensity score-matched external control from the Enroll-HD natural history database. uniQure said the FDA and company aligned on a confirmatory study design: a randomized standard-of-care trial in about 200 patients, randomized 2:1 to ifezuntirgene inilparvovec or standard of care, with Total Functional Capacity as the primary endpoint at 36 months. Patient screening in the U.S. is planned by the end of 2026.

The company said the Phase I/II program has dosed 51 patients across two multi-center studies and additional cohorts, including U.S. randomized and European open-label cohorts. The therapy has received FDA Breakthrough Therapy, RMAT, and Fast Track designations.