The FDA has accepted uniQure’s application for its experimental Huntington’s disease gene therapy, AMT-130 (ifezuntirgene inilparvovec), and granted it Priority Review.
The FDA’s target action date is May 1, 2027. Priority Review speeds the review timetable; it does not guarantee approval.
The application is being considered under the Accelerated Approval pathway. The FDA said it is tentatively planning an advisory committee meeting, but has not set a date.
If approved, AMT-130 could become the first disease-modifying treatment for Huntington’s disease. The therapy is designed to reduce production of the huntingtin protein by delivering a gene-silencing treatment directly into the brain.
UniQure says it has aligned with the FDA on key elements of a planned confirmatory study of about 200 patients. The company plans to begin screening by the end of 2026; the study’s primary measure is total functional capacity at 36 months.
The milestone follows a difficult regulatory period for the company. The FDA’s decision on the application remains pending, and an advisory committee discussion could add scrutiny before the target date.
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