uniQure N.V. (QURE) said the feedback came during a recent Type B meeting with the agency, where regulators confirmed that the three-year analysis from its Phase I/II study would be acceptable as the primary foundation for a BLA submission for AMT-130, its gene therapy for Huntington's disease. The company now plans to submit the application in the third quarter of 2026. A closer look at the FDA guidance
The FDA also asked to finalize the design of the required confirmatory study before the BLA is filed, including the potential use of concurrent controls on standard-of-care therapy instead of a sham procedure—a notable shift in trial expectations for this rare neurodegenerative disorder. Regulators emphasized they would work "as expeditiously as possible" with uniQure to align on the study design.
CEO Matt Kapusta said the agency's position reflects "the outcome we have worked toward" and highlighted the FDA's commitment to addressing the unmet needs of patients living with Huntington's disease. He added that the strength and consistency of the clinical data give the company confidence in AMT-130's potential impact.
About AMT-130 and the clinical program
AMT-130 has received RMAT, Breakthrough Therapy, and Fast Track designations—the first RMAT designation ever granted for Huntington's disease—reflecting the therapy's potential significance for a condition with no approved disease-modifying treatments.
The Phase I/II program includes two multi-center, dose-escalating studies evaluating safety, tolerability, and exploratory efficacy. Based on prior FDA interactions, data from the first two cohorts may be compared to the Enroll-HD natural history dataset under a prespecified statistical plan, which could serve as the primary basis for the BLA.
uniQure expects to receive final meeting minutes from the FDA within 30 days, which will guide the final steps toward submission.
QURE has traded between $8.73 and $71.50 over the past year. The stock is currently trading at $47.00, up 74.47%.
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June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.