The Food and Drug Administration has accepted for review uniQure’s biologics license application for ifezuntirgene inilparvovec, also called AMT-130, an investigational gene therapy for Huntington’s disease filed under the accelerated approval pathway, and granted it priority review with a target action date of May 1, 2027. In its acceptance letter the agency said it tentatively plans to discuss the application at an advisory committee meeting, with the date still to be set. The application rests on 36 month Phase I/II results compared with a matched external control group from the Enroll-HD natural history study, and a confirmatory trial of about 200 patients is expected to start screening by the end of 2026.
FDA accepts uniQure Huntington's gene therapy application for review
The FDA has accepted uniQure's application for ifezuntirgene inilparvovec, a gene therapy for Huntington's disease, granted it priority review, and set a decision date of May 1, 2027.
Why it matters: If approved under the accelerated pathway, the therapy would be the first treatment that modifies the course of Huntington's disease, a condition with no disease modifying option today.
Data as of uniQure announcement of November 2, 2026, read in full in this run, and the Endpoints News briefing of November 2, 2026. Acceptance and priority review are the company's report of the FDA's letter; the agency has not published its own release. Developing item.
Sources
- uniQure announces FDA acceptance and priority review of the Biologics License Application for ifezuntirgene inilparvovec for the treatment of Huntington's disease - uniQure, via GlobeNewswire - 2026-11-02
- UniQure gets review date and adcomm; Inovio's stock surges after approval - Endpoints News - 2026-11-02