uniQure says FDA announced Phase I/II AMT-130 data unlikely to support BLA

uniQure (QURE) announced that the company received final meeting minutes from the U.S. Food and Drug Administration, FDA, regarding a pre-Biologics License Application, BLA, meeting held on October 29, 2025 to discuss the application for AMT-130, an investigational gene therapy for Huntington’s disease, HD. In the final meeting minutes, and consistent with uniQure’s November 3, 2025 press release, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission. uniQure is carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026.