FDA places uniQure’s Huntington’s gene therapy in priority review, targeting May 2027 approval

The U.S. Food and Drug Administration has accepted uniQure’s filing for its Huntington’s disease gene therapy and placed the product under priority review, a step that could accelerate a decision by May 1, 2027.

The agency also indicated it may hold an advisory committee meeting to discuss the therapy, and the company said it has aligned with the FDA on key elements of a confirmatory trial.

The planned trial will enroll about 200 patients, randomizing them in a 2-to-1 ratio to receive the gene therapy or standard of care, with the primary endpoint measuring total functional capacity at 35 months.

uniQure intends to begin patient screening by the end of 2026 so the study can be underway when a regulatory decision is made. The therapy, called AMT-130 (ifezuntirgene inilparvovec), delivers a micro-RNA to silence the huntingtin gene directly into the brain through small skull openings.

The company has previously faced regulatory scrutiny, but this acceptance marks a significant milestone in its effort to bring a disease-modifying treatment for Huntington’s disease to market.