uniQure announced that the FDA accepted its Biologics License Application for ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for Huntington's disease, and granted Priority Review with a target action date of May 1, 2027. The application seeks accelerated approval using 36-month Phase I/II data compared with a propensity-score-matched external control from the Enroll-HD natural-history database. The FDA tentatively plans an advisory committee meeting, while uniQure has aligned with the agency on key elements of a confirmatory randomized study of approximately 200 patients. Patient screening for that study is expected to begin by the end of 2026.
SEC
Primary source: uniQure SEC disclosure, November 2
Why it matters strategically
The application represents a consequential test of regulatory flexibility in serious rare diseases where conventional randomized trials can be difficult.
The FDA has accepted the application for review, but acceptance and Priority Review do not establish that the evidence is sufficient for approval.
The central scientific question is whether the external control reliably estimates the disease progression that would have occurred without treatment.
