Regulatory timeline: Huntington’s disease gene therapy moves toward potential approval date

The FDA set a May 1, 2027 Prescription Drug User Fee Act action date for uniQure’s Huntington’s disease gene therapy candidate ifezuntirgene inilparvovec (AMT-130), following acceptance of its BLA under Priority Review. The agency also indicated it is tentatively planning to discuss the submission at a future advisory committee meeting. uniQure said the acceptance reverses earlier regulatory opposition and marks a key milestone for the Huntington’s disease community, while an analyst noted it reduces regulatory risk. Still, investors appeared cautious, with uniQure shares dipping after the news. The decision calendar keeps pressure on uniQure to demonstrate durable disease modification as it prepares for potential adcomm scrutiny and final FDA action next year.