Nov 2, 2026 —
The FDA has accepted uniQure’s biologics license application for AMT-130 (ifezuntirgene inilparvovec), its gene therapy for Huntington’s disease, granting Priority Review and setting a target action date of May 1, 2027, under the accelerated approval pathway. An advisory committee meeting is expected. If approved, it would be the first treatment shown to slow the course of this fatal, inherited neurodegenerative disease.
AMT-130 is a one-time adeno-associated virus (AAV) gene therapy, built on an AAV5 vector and delivered by MRI-guided infusion directly into the striatum, deep in the brain. It carries a microRNA designed to lower production of the mutant huntingtin protein that drives the disease.
The filing rests on a small, externally controlled dataset. In an updated 36-month analysis of 15 high-dose patients, uniQure reported an 80% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale versus a matched natural-history control, a nominal p=0.005, with cerebrospinal-fluid neurofilament light, a marker of neuronal damage, holding near baseline. A longer 48-month look at the first 12 patients showed a 44% slowing that was not statistically significant. A roughly 200-patient randomized confirmatory trial is planned.
