When will the FDA approve uniQure's AMT-130 for Huntington's disease?

My median prediction for FDA approval of AMT-130 is 26th January 2028 because uniQure's four-year data showed a 44% slowing in disease progression without achieving statistical significance.

uniQure submitted a Biologics License Application on 2 September 2026 seeking accelerated approval of AMT-130 (ifezuntirgene inilparvovec), a one-time AAV gene therapy delivered by neurosurgery, for Huntington's disease. The application relies on comparisons with an external natural-history control, an approach on which the FDA's position has shifted during development. The FDA's decision on whether to accept the filing is due around early November 2026; with priority review an action date would fall around the second quarter of 2027. AMT-130 would be the first disease-modifying treatment approved for Huntington's disease. CBER also decides Capricor's deramiocel (PDUFA 22 November 2026), another therapy filed on contested evidence.

Resolves on the date the US FDA approves AMT-130 (accelerated or full approval) for Huntington's disease, as shown by an FDA announcement, the approval letter or a uniQure press release. A refusal to file or a Complete Response Letter does not resolve the question; a later approval does.