uniQure says FDA no longer agrees AMT-130 data ‘adequate’ to support BLA

uniQure (QURE) received feedback from the U.S. Food and Drug Administration during a recent pre-Biologics License Application meeting regarding AMT-130, an investigational gene therapy for Huntington’s disease. Though final meeting minutes have not yet been received, based on the discussions at the meeting, uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission. This is a key shift from prior communications with the FDA in multiple Type B meetings over the past year. Consequently, the timing of the BLA submission for AMT-130 is now unclear. uniQure expects to receive final minutes within 30 days of the meeting and plans to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130. The FDA granted AMT-130 Breakthrough Therapy designation based upon data from the Phase I/II studies compared to external controls in April 2025 and Regenerative Medicines Advanced Therapy designation in May 2024. “We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway,” said Matt Kapusta, CEO. “This news is unexpected, and we are truly disappointed for people living with HD, who have no disease-modifying treatment options for this devastating disease. We strongly believe that AMT-130 has the potential to bring substantial benefit to patients, and we remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.” In addition to continuing to partner with the FDA on progressing AMT-130 for the treatment of Huntington’s disease, uniQure plans in parallel to progress discussions with other regulatory agencies, including in the European Union and United Kingdom.