On March 2, 2026, uniQure announced that the FDA deems Phase 1/2 data from its single-arm trial of AMT-130 insufficient for marketing approval, recommending a prospective, randomized, double-blind, sham surgery-controlled study12345
AMT-130 is an investigational one-time gene therapy delivered via brain surgery to lower mutant huntingtin protein levels in Huntington's disease patients235
Following a January 30, 2026 Type A meeting, uniQure plans to request another FDA meeting in Q2 2026 to discuss Phase 3 study designs and alternative paths1345
FDA Commissioner Marty Makary appeared to criticize AMT-130 in a recent CNBC interview, referencing skull-drilling procedures without clear benefit4
Despite positive three-year data showing slowed disease progression, analysts note FDA's stance contrasts with promised flexibility for rare diseases346
Sources:
1. https://www.statnews.com/2026/03/02/uniqure-huntingtons-disease-fda-blocked/
2. https://en.hdbuzz.net/the-road-ahead-for-uniqure-fda-says-more-data-needed-for-amt-130-gene-therapy/
3. https://www.biopharmadive.com/news/uniqure-fda-meeting-trial-requirement-huntingtons-gene-therapy/813488/
4. https://www.biospace.com/fda/uniqure-eyes-phase-3-for-huntingtons-gene-therapy-despite-makarys-apparent-criticism
5. https://globalgenes.org/raredaily/fda-wants-additional-study-from-uniqure-for-huntingtons-disease-gene-therapy/
6. https://www.bioworld.com/articles/729268-uniqure-sinks-as-fda-demands-new-trial-for-huntingtons-gene-therapy