FDA Accepts uniQure's Huntington's Gene Therapy BLA for Priority Review, Sets May 1, 2027 Decision Date

QURE announced on November 2 that the U.S. Food and Drug Administration accepted for review its Biologics License Application for ifezuntirgene inilparvovec, also known as AMT-130, an investigational gene therapy for Huntington's disease submitted under the Accelerated Approval pathway. The application received Priority Review with a PDUFA target action date of May 1, 2027, and in its acceptance letter the FDA said it is tentatively planning to discuss the application at an Advisory Committee meeting, with dates still to be set.

The filing is built on 36-month data from the Phase I/II study, compared against a propensity score-matched external control drawn from the Enroll-HD natural history database, according to the company's release. uniQure said that if approved, the therapy would be the first disease-modifying treatment for Huntington's disease, a condition affecting about 75,000 people in the U.S., EU and UK with no approved therapy to delay onset or slow progression. The program already holds Breakthrough Therapy, RMAT and Fast Track designations.

The evidence base is the key debate. The data come from a small Phase I/II program rather than a randomized pivotal trial, and BioPharm International reported in September that the 48-month cUHDRS measure showed 44% slowing of progression but did not reach statistical significance (p=0.144). Accelerated approval would require confirmation: uniQure plans a randomized trial of about 200 patients, assigned 2:1 to treatment or standard of care with Total Functional Capacity at 36 months as the primary endpoint, and expects to begin U.S. screening by the end of 2026.

What to watch: the date and outcome of the FDA Advisory Committee meeting, the May 1, 2027 PDUFA action date, and the start of the confirmatory trial.