The US Food and Drug Administration (FDA) has accepted an application for an experimental gene therapy for Huntington's disease for priority review, with a decision expected by 1 May 2027.
The FDA has agreed to review the application under its accelerated approval pathway. It has also indicated that an advisory committee meeting is being considered to discuss the application. If approved, the therapy would become the first disease-modifying treatment for Huntington's disease, an inherited neurodegenerative condition that causes progressive movement difficulties, cognitive decline and behavioural changes.
The application is supported by data from phase 1/2 clinical trials (see BioNews 1344). However, four-year follow-up results from 12 high-dose participants showed a 44 percent slowing of progression was not statistically significant (see BioNews 1360).
UniQure has also submitted an application for approval to the UK's Medicines and Healthcare products Regulatory Agency, which validated the application in the third quarter of 2026.
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