FDA grants uniQure's Huntington's gene therapy Priority Review on small, externally ...

The US FDA has accepted uniQure's gene therapy for Huntington's disease, ifezuntirgene inilparvovec (AMT-130), for Priority Review with a PDUFA target date of May 1, 2027, under the Accelerated Approval pathway. This therapy could become the first disease-modifying treatment for Huntington's disease, based on data showing slower functional decline in treated patients, pending a confirmatory trial aligned with the FDA's requirements.

This Wire brief sits within Fusion42's coverage of Biotech, and 3 sources have reported it between 2 Nov 2026 and 3 Nov 2026.