FDA accepts uniQure's Huntington's gene therapy AMT-130 for priority review, May 1 action date
- The FDA accepted uniQure's biologics license application, filed September 2, for AMT-130 (ifezuntirgene inilparvovec) in Huntington's disease, with priority review and a May 1 action date.
- The agency said it tentatively plans to take the application to an advisory committee. Approval would make it the first disease-modifying Huntington's therapy.
- The filing follows a 2025 FDA reversal on data adequacy and a later policy shift. A confirmatory trial of about 200 patients, with a 36-month functional capacity endpoint, is expected to start screening by end-2026.
💡 Points Based on public reports
Priority review plus a tentative advisory committee suggests the agency wants outside scrutiny rather than a quiet sign-off, which fits a filing that follows a year of shifting positions on data adequacy. The next tells are how the panel reads the data and whether any accelerated approval is tied to the 200-patient confirmatory trial; that will set expectations for other CNS gene therapies.
UniQure snags May Day decision date for Huntington's gene therapy after arduous year ↗