uniQure Wins Priority Review For AMT-130, Setting May 1, 2027 Huntington’s Decision

uniQure has cleared a regulatory hurdle that looked uncertain only months ago, with the FDA accepting its application for Huntington’s disease gene therapy ifezuntirgene inilparvovec, also called AMT-130, under priority review. The agency set a May 1, 2027 decision date, and uniQure said the FDA is tentatively planning to discuss the application at an advisory committee meeting.

The acceptance is a meaningful signal for a program that has swung between optimism and regulatory resistance. uniQure said the submission was based on the 36-month analysis it shared in September 2025, when the higher dose showed a statistically significant 75% slowing of disease progression. More recently, the company reported a 48-month analysis showing a 44% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale, but that result fell short of statistical significance.

The road here

AMT-130 has had a difficult path to this point. In 2022, dosing was paused for two groups in an open-label, European phase 1/2 trial after three patients were hospitalized with various symptoms; the pause was later lifted in November 2022.

Regulatory alignment was also unstable. In September 2025, despite the positive 36-month phase 1/2 update, the FDA no longer agreed that those data were enough to support an approval filing and had previously demanded data from another trial. The agency changed course again in June 2026, giving uniQure the go-ahead to file for accelerated approval.

That sequence makes the current review more than an administrative milestone. It suggests the FDA is prepared to formally assess whether the earlier dataset can support approval, even though later follow-up weakened the statistical picture and the agency had earlier pushed for more traditional evidence.

What Comes Next

uniQure said it aligned with the FDA on certain key elements of a confirmatory trial. The study will enroll 200 patients randomized 2-to-1 to receive AMT-130 or standard of care, with total functional capacity measured at 35 months as the primary endpoint. The company plans to start patient screening by the end of 2026 so the study is already underway by the time of an approval decision.

AMT-130 uses an miRNA designed to silence the huntingtin gene, delivered directly into the brain through small holes in the skull. If approved, it would move a one-time gene therapy into a disease area that has waited decades for a treatment intended to change disease course.