uniQure N.V. (NASDAQ: QURE) announced on November 2, 2026, that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for ifezuntirgene inilparvovec. The application, submitted under the Accelerated Approval pathway, has been granted Priority Review status. The Prescription Drug User Fee Act (PDUFA) target action date for the FDA’s decision is set for May 1, 2027.
The company stated that the FDA has tentatively planned to discuss the application at an Advisory Committee meeting, though the specific date and timeline have not yet been finalized. The BLA is supported by a 36-month data analysis from the company's Phase I/II clinical study, which compared the treatment to a propensity score-matched external control derived from the Enroll-HD natural history database.
uniQure and the FDA have aligned on the design of a confirmatory study. This trial will be a randomized, standard-of-care study enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care. The primary endpoint of the study is Total Functional Capacity (TFC) at 36 months. The company plans to initiate patient screening in the U.S. by the end of 2026.
Ifezuntirgene inilparvovec is the first investigational therapy for Huntington’s disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as Fast Track designation. The treatment utilizes a proprietary gene-silencing miQURE platform and is administered via a single MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum.
