uniQure Files First-Ever Huntington's Gene Therapy Application After FDA Reversal

uniQure has submitted a Biologics License Application for its one-time gene therapy AMT-130, asking the FDA for accelerated approval on the strength of 3-year data the agency had rejected as insufficient six months earlier.

uniQure said on September 2, 2026 that it had submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration seeking accelerated approval of ifezuntirgene inilparvovec, known as AMT-130, a one-time gene therapy delivered directly into the brain of people with Huntington's disease. The company has asked the FDA for priority review, which, if granted, would shorten the agency's review to roughly eight months after the initial 60-day filing check. It is the first marketing application ever filed for a gene therapy in Huntington's disease, according to uniQure.

The filing follows a reversal by the FDA. In March 2026, the agency told uniQure in writing that data from its Phase I/II studies, measured against an external comparison group rather than a concurrent control, could not by itself support a marketing application, and recommended a randomized, sham-surgery-controlled trial, a demand similar to the one that recently led Passage Bio to close its frontotemporal dementia gene therapy trial to new patients. Three months later, following further meetings, the FDA told uniQure the opposite: that the same three-year Phase I/II analysis, compared with a propensity-matched external control drawn from the Enroll-HD natural history database, could serve as the primary basis for a BLA under the accelerated approval pathway.

The application rests on 36-month data from a small trial. In the high-dose group, uniQure reported a 75 percent slowing of decline on a composite Huntington's rating scale relative to the external control (p=0.003), and a 60 percent slowing on a functional capacity measure (p=0.033), alongside an 8.2 percent reduction from baseline in a cerebrospinal fluid marker of nerve cell damage. uniQure said no new drug-related serious adverse events had been recorded since December 2022. The company has not yet published the full dataset in a peer-reviewed journal.

The FDA has not yet decided whether to accept the application for review, and it has separately asked uniQure to agree on the design of a required confirmatory study, using a standard-of-care control rather than sham surgery, before any approval would take effect. Analysts tracking the filing have noted that regulatory risk remains given recent turnover in FDA leadership. If accelerated approval is eventually granted, it would be conditional: the therapy could be withdrawn if the confirmatory study does not later confirm a clinical benefit.