FDA to review uniQure's Huntington's gene therapy, decision due May 1, 2027
QURE•The FDA accepted uniQure's application for its Huntington's disease gene therapy AMT-130 and set a decision date of May 1, 2027. The agency granted a faster review and is considering an outside-expert meeting, the company said.
1. FDA accepts application
The FDA accepted uniQure's application for AMT-130 and is reviewing it under the accelerated approval pathway. The agency set May 1, 2027, as its decision date and is considering holding a meeting of outside experts to discuss the application.
2. Evidence and follow-up trial
The application draws on early- to mid-stage study results comparing treated patients with a group from an external Huntington's disease database. UniQure said it has treated 51 patients across its studies and plans to begin US patient screening by year-end for a 200-patient follow-up trial, with two-thirds receiving the gene therapy and the rest standard care.
3. Earlier trial results
The review follows uniQure's report months ago that AMT-130 failed to slow disease progression in a four-year trial. The FDA later reversed course after a senior agency official described the treatment as a “failed product.”
