Huntington's Disease Gene Therapy AMT-130 Is on Track for FDA Approval After uniQure Reaches Regulatory Agreement

For people living with Huntington's disease — and for the families watching it take its relentless toll — June 17, 2026, may eventually be remembered as the day the trajectory of the disease changed.

uniQure N.V. announced that morning that during a recent Type B meeting with the FDA, the agency confirmed that three-year data from the Phase I/II study of AMT-130 — its gene therapy for Huntington's disease — would be acceptable as the primary basis for a Biologics License Application seeking accelerated approval. The company intends to submit the BLA in the third quarter of 2026.