UniQure's AMT-130 Gene Therapy for Huntington's Disease: FDA Unlikely to Approve Based on Current Data

UniQure received final meeting minutes from the FDA regarding its pre-Biologics License Application for AMT-130, a gene therapy for Huntington's disease. The FDA stated that data from Phase I/II studies are unlikely to support a BLA submission. UniQure plans to request a follow-up meeting with the FDA in Q1 2026 to collaborate on advancing AMT-130. The company remains committed to collaborating with the FDA to bring the treatment to patients as rapidly as possible.