uniQure Says FDA Grants Priority Review to Huntington’s Gene Therapy

uniQure said Monday the U.S. Food and Drug Administration (FDA) accepted its biologics license application for investigational Huntington’s disease gene therapy ifezuntirgene inilparvovec, also known as AMT-130, and granted Priority Review. The agency set May 1, 2027, as the target action date.

The application seeks accelerated approval. uniQure said the FDA’s acceptance letter tentatively proposed an advisory committee discussion, but no meeting date or timeline has been set. The company submitted the application on Sept. 2, requesting Priority Review; the May 1 date is a target for FDA action, not an approval.

The BLA is supported by an earlier 36-month Phase I/II analysis comparing 12 high-dose patients with matched external controls from the ENROLL-HD natural-history database. In September, uniQure said the FDA had communicated that 36-month data from 12 high-dose patients could serve as the application’s primary basis.

A Sept. 29 update reported later, 48-month results and was issued after the BLA submission, so it was not included in the application. In that update, the cUHDRS measure showed 44% slowing compared with external controls, a result that was not statistically significant (p=0.144). Total Functional Capacity (TFC) showed 61% slowing, with a nominal p-value of 0.008. Follow-up data were missing for 53% of the updated matched controls at 48 months; uniQure said this could have understated disease progression in the control group.

uniQure said it and the FDA aligned on key elements of a confirmatory study. The planned trial would enroll about 200 patients randomized 2:1 to the therapy or standard of care, with TFC at 36 months as the primary endpoint. The company plans to begin U.S. screening by the end of 2026.

AMT-130 is a one-time gene therapy administered through MRI-guided neurosurgery into the striatum. uniQure CEO Matt Kapusta called the FDA acceptance an “important milestone for the Huntington’s disease community.”