Investing.com -- uniQure N.V. (NASDAQ:QURE) stock fell 14% after the gene therapy company received feedback from the U.S. Food and Drug Administration (FDA) indicating that current data for its Huntington’s disease treatment is unlikely to support approval.
The FDA conveyed in final meeting minutes from an October 29 pre-Biologics License Application meeting that data from Phase I/II studies of AMT-130 would probably not provide sufficient primary evidence to support a BLA submission. This feedback confirms what uniQure had previously disclosed in a November 3 press release.
uniQure is currently evaluating the FDA’s feedback and plans to request a follow-up meeting with the regulator in the first quarter of 2026 to discuss next steps for the investigational gene therapy.
"We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible," said Matt Kapusta, chief executive officer at uniQure. "The support we have seen these last weeks from the Huntington’s disease community, including patients, families, caregivers, clinicians and advocates reinforces the urgency of the unmet need in Huntington’s disease."
AMT-130 is being developed as a potential treatment for Huntington’s disease, a rare genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to movement, cognitive, and psychiatric disorders.
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