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A gene therapy for Huntington's disease just got a major regulatory boost. The FDA has granted Priority Review to uniQure's ifezuntirgene inilparvovec (AMT-130), setting a decision deadline of May 1, 2027. If approved, it could become the first treatment to actually slow the progression of this devastating inherited brain disease.
A gene therapy for Huntington's disease just got a major regulatory boost.
The FDA has accepted uniQure's biologics license application (BLA) for ifezuntirgene inilparvovec (AMT-130) and granted it Priority Review, with a target action date of May 1, 2027. Submitted under the accelerated approval pathway, the therapy uses uniQure's gene-silencing miQURE platform to deliver a microRNA that suppresses the mutant huntingtin gene — administered as a one-time, MRI-guided neurosurgical injection directly into the brain's striatum. It already holds Breakthrough Therapy, RMAT, and Fast Track designations, making it the first Huntington's candidate to hold both Breakthrough Therapy and RMAT status simultaneously.
The BLA is anchored by 36-month Phase I/II data from 51 patients, which showed the high-dose group experienced 75% slowing of disease progression on the primary endpoint (cUHDRS) compared to an external natural history control. Huntington's disease affects an estimated 75,000 people across the US, EU, and UK, and currently has no approved therapy that delays onset or slows progression.
By the Numbers:
Why it matters: Huntington's disease is a fatal, inherited condition with no disease-modifying treatment — patients and families have waited generations for a therapy that changes its course. A successful approval would mark a historic first and validate gene silencing as a viable approach for neurodegenerative diseases.
