On March 2, 2026, uniQure announced FDA meeting minutes stating Phase 1/2 data for AMT-130 are insufficient for approval1.
FDA official stated agency 'not convinced' of therapeutic benefit and requires a new randomized, sham-controlled Phase 3 trial23.
Sham procedure involves anesthesia and 1-3 scalp nicks, not full burr holes as claimed by uniQure's CMO3.
uniQure disputes FDA's prior guidance and plans a Type B meeting in Q2 2026 to discuss Phase 3 designs14.
AMT-130 is an AAV-delivered microRNA gene therapy to lower huntingtin protein in the brain for Huntington's disease1.
Sources:
1. https://en.hdbuzz.net/the-road-ahead-for-uniqure-fda-says-more-data-needed-for-amt-130-gene-therapy/
2. https://www.statnews.com/2026/03/03/uniqure-huntingtons-treatment-fda-official-comments/
3. https://www.biospace.com/fda/fda-official-fires-back-at-uniqure-doubles-down-on-sham-controlled-trial
4. https://www.fiercebiotech.com/biotech/pressure-builds-back-and-forth-between-uniqure-and-fda-intensifies