UniQure announced that the FDA has accepted the marketing application for AMT-130 gene therapy to treat Huntington’s disease, with an expected decision date of May 1, 2027, and an advisory committee review is tentatively planned.

If approved, ifezuntirgene inilparvovec would be the first disease-modifying treatment for Huntington’s disease and has FDA Breakthrough Therapy and RMAT designations, as well as Fast Track designation.

The FDA accepted for review and granted Priority Review to the Biologics License Application for ifezuntirgene inilparvovec, with a PDUFA target action date of May 1, 2027.

In September 2026, four-year data were weaker than expected, contributing to stock declines, but the company emphasized the importance of the ongoing regulatory process.

Analysis of the mitochondrial replacement topic is behind a STAT+ paywall, limiting detailed public reporting and context.

The press release includes forward-looking statements and outlines regulatory, confirmatory data, and post-approval risks.

The Phase I/II program dosed 51 patients across multiple cohorts, including a U.S. randomized study and a European open-label study, with additional cohorts exploring dose combinations and immunosuppression.

UniQure provides investor and media contact details and references clinical trial identifiers NCT05243017 and NCT04120493.

Earlier in 2025, the FDA reversed a prior stance after three-year data, saying the data were not adequate primary evidence for a BLA submission, contributing to a volatile regulatory path.

The confirmatory trial remains a key priority, with screening expected to begin by year-end and about 200 patients to be enrolled, comparing AMT-130 to standard of care over 36 months to assess total functional capacity.

The BLA relies on 36-month Phase I/II data using a propensity score-matched external control, with a planned confirmatory trial of about 200 patients in a 2:1 randomization, primary endpoint Total Functional Capacity at 36 months.

Overall development has been rocky, but regulatory momentum centers on the May 1 decision date and potential advisory committee discussion.