uniQure N.V. (NASDAQ:QURE) shares plunged 75% on Monday after the biotech company announced a major regulatory setback for its experimental Huntington’s disease gene therapy, AMT-130.
The Netherlands-based firm revealed that the U.S. Food and Drug Administration (FDA) no longer agrees that data from Phase I/II trials, when compared with external control datasets, would be sufficient to support a Biologics License Application (BLA). The change marks a sharp departure from the agency’s previous stance over the past year.
uniQure described the FDA’s feedback as “unexpected” and “a drastic change” from its prior guidance issued in November 2024, when regulators had indicated that data from ongoing studies using natural history external controls could form the primary basis for a BLA submission under the Accelerated Approval pathway.
“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,” said Matt Kapusta, uniQure’s chief executive officer.
The company stated that the timeline for submitting a BLA is now uncertain but expects to receive final meeting minutes within 30 days and intends to “urgently interact with the FDA” to identify a viable path forward for AMT-130’s accelerated approval.
Despite the setback, uniQure emphasized that AMT-130 still holds Breakthrough Therapy designation (granted in April 2025) and Regenerative Medicine Advanced Therapy (RMAT) status (received in May 2024) from the FDA.
uniQure added that it will continue discussions with regulators in the European Union and United Kingdom, while also engaging with the FDA to determine next steps for advancing AMT-130 in the United States.