FDA Gives uniQure’s Huntington’s Therapy Priority Review
The FDA accepted uniQure’s application for review and set a May 1, 2027 target date for action on the gene therapy.
Key takeaways
- The FDA accepted uniQure’s application and granted it Priority Review.
- The FDA set a May 1, 2027 target action date.
- uniQure plans to begin U.S. patient screening for a confirmatory trial by the end of 2026.
The FDA accepted uniQure’s application for its experimental Huntington’s disease gene therapy and granted it Priority Review. The agency set May 1, 2027, as its target date for action.
The application was submitted under the Accelerated Approval pathway. The FDA said it is tentatively planning to discuss the application at an advisory committee meeting, but has not finalized the date or timing.
The company said the application is supported by 36-month data from a Phase I/II study. UniQure plans to begin U.S. screening for a confirmatory trial by the end of 2026.
The planned trial would enroll about 200 patients, with two assigned to the therapy for every one assigned to standard care. Its primary endpoint is Total Functional Capacity at 36 months.
This article was produced with the help of AI technology. Based on reporting by the outlets listed above.
