FDA grants uniQure Huntington’s gene therapy priority review

The FDA accepted uniQure’s application for its investigational Huntington’s disease gene therapy, ifezuntirgene inilparvovec (AMT-130), and granted it Priority Review under the Accelerated Approval pathway, setting a target decision date of May 1, 2027. The agency is tentatively planning an advisory committee meeting. The application is supported by 36-month Phase I/II results compared with an external natural-history control; acceptance starts the FDA’s review but does not mean the treatment is approved or that the evidence will meet the agency’s standard. AMT-130 is a one-time, MRI-guided neurosurgical treatment intended to silence the huntingtin gene. uniQure and the FDA have aligned on a randomized confirmatory study of about 200 patients, with the company planning to begin U.S. screening by the end of 2026.