uniQure Submits BLA to FDA for AMT-130 (Ifezuntirgene Inilparvovec) AAV Gene Therapy for Huntington's Disease Under Accelerated Approval Pathway
uniQure N.V. (NASDAQ: QURE) announced Wednesday September 2, 2026 the submission of a Biologics License Application (BLA) to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational AAV-delivered gene therapy for the treatment of Huntington's disease. The BLA is the first ever submitted for a Huntington's disease therapy of any kind. AMT-130 is delivered by stereotactic intraparenchymal injection into the striatum and expresses a microRNA designed to silence the mutant huntingtin (mHTT) gene. The submission is supported by three-year data from the Phase 1/2 program compared to a propensity score-matched external control from the Enroll-HD natural history database. uniQure also submitted a Marketing Authorisation Application (MAA) to the UK MHRA on the same day. A four-year data readout from the ongoing Phase 1/2 study is expected before end of Q3 2026. AMT-130 previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Approval would establish the first disease-modifying therapy for Huntington's, where standard-of-care remains symptom management with tetrabenazine and deutetrabenazine plus antipsychotics.