The FDA has accepted uniQure’s application for its Huntington’s disease gene therapy under priority review, alongside a tentative plan to convene an advisory committee meeting.
Despite a tumultuous recent run with the FDA, uniQure believes that an approval by May 1, 2027, would be a “defining milestone.”
In accepting the filing, the FDA stated that they tentatively plan to discuss it at an advisory committee meeting, uniQure said in its Nov. 2 release.
UniQure said it also aligned with the FDA on certain key elements of a confirmatory trial. The study will enroll 200 patients to be randomized 2-to-1 to receive either the company's gene therapy or standard of care. The primary endpoint will measure total functional capacity at 35 months.
To ensure that the study is already underway by the time of an approval decision, uniQure said it plans to start patient screening by the end of 2026.
UniQure’s Huntington’s disease therapy, ifezuntirgene inilparvovec (AMT-130), incorporates a miRNA designed to silence the huntingtin gene by delivering it directly into the brain through small holes in the skull.
The company has had a hard time with the FDA before reaching this regulatory milestone of submission acceptance.
In 2022, AMT-130 was scrutinized after safety signals prompted a pause in dosing for two groups in an open-label, European phase 1/2 trial. The pause, which was recommended by a safety monitoring board after three patients were hospitalized with various symptoms, was lifted in November 2022.
Three years later, in September 2025, uniQure reported positive phase 1/2 data showing that patients who received the higher dose of AMT-130 showed a statistically significant 75% slowing of disease progression at the 36-month mark. UniQure was met with contention shortly after, as the FDA no longer agreed that the data from that study were enough to support an approval filing.
The FDA changed its mind again in June 2026, giving the biotech the go-ahead to file for accelerated approval after previously demanding data from another trial. Most recently, uniQure reported that at the 48-month analysis from the phase 1/2 trial, AMT-130 showed a 44% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale, falling short of statistical significance and crashing the stock 41%.
The FDA submission “was based on the 36-month analysis publicly shared in September 2025,” a uniQure spokesperson told Fierce.
“For uniQure, approval would be a defining milestone. It would validate our years of commitment to the [Huntington’s disease] community and mark our transition to bringing a one-time treatment to patients who have waited decades for meaningful progress,” the company said.