uniQure Faces Regulatory Setback for AMT-130 in Huntington’s Disease

LEXINGTON, Mass. and AMSTERDAM, Nov. 3, 2025 (GLOBE NEWSWIRE) — uniQure (NASDAQ: QURE) has announced a significant update regarding its investigational gene therapy AMT-130 for Huntington’s disease. During a pre-Biologics License Application (BLA) meeting, the U.S. Food and Drug Administration (FDA) indicated a shift in its stance on the adequacy of Phase I/II trial data against an external control.

uniQure stated that the FDA no longer agrees that the earlier Phase I/II data meets the criteria needed as primary evidence for a BLA submission. This marks a departure from the guidance received in prior meetings, creating uncertainty around the submission timeline for AMT-130.

The company expects to receive the final minutes from the meeting within 30 days and plans to urgently engage with the FDA to clarify the path forward. In addition, uniQure will also be communicating with regulatory authorities in the European Union and the United Kingdom.

AMT-130 has previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations due to its potential benefits in treating patients with Huntington’s disease. “We are surprised by the FDA’s feedback, which is a drastic change from earlier indications,” said a company spokesperson. “This news is disappointing for those affected by Huntington’s disease.”

uniQure remains committed to working collaboratively with the FDA to explore options for accelerating the approval process for AMT-130. The FDA’s concerns add to the complexity of the regulatory landscape for this promising therapy, which aims to provide a viable treatment option for patients with no currently available disease-modifying therapies.