UniQure to seek approval of Huntington’s gene therapy after FDA U-turn

UniQure confirmed it will submit its AMT-130 gene therapy for regulatory review for Huntington's disease after the US Food and Drug Administration (FDA) rescinded previous demands for additional clinical data. Huntington's disease is an inherited neurodegenerative disorder affecting approximately 75,000 people in the US, Europe, and the UK, and is fatal within 15 to 20 years of symptom onset. AMT-130 would be the first therapy specifically approved for this disease. The FDA initially rejected UniQure's submission in November 2024 citing insufficient data from a Phase I/II trial, but changed its position following a January meeting. UniQure plans to submit a biologics License Application (BLA) in Q3 2026 and will need to conduct a confirmatory study. The gene therapy AMT-130 is delivered directly into the brain through a single neurosurgical procedure and acts in the striatum, the brain region most damaged in Huntington's disease.