The US Food and Drug Administration says early trial data for a Huntington’s gene therapy are not sufficient to support a marketing application.
Gene therapy company uniQure said final meeting minutes from a Type A meeting with the FDA on 30 January 2026 confirmed the regulator’s position on AMT-130.
AMT-130 is an investigational gene therapy for Huntington’s disease, a rare inherited neurodegenerative disorder that gradually damages brain cells and is ultimately fatal.
The FDA said it could not agree that data from phase I/II studies, compared with an external control group, provided the primary evidence of effectiveness required to support a marketing application.
The regulator strongly recommended that uniQure conduct a prospective, randomised, double-blind, sham surgery-controlled study. In this type of trial, some participants undergo a simulated surgical procedure to act as a comparison group.
uniQure said it plans to continue discussions with the FDA regarding phase III development considerations and intends to request a Type B meeting in the second quarter of 2026 to discuss possible study design approaches.
“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said Matt Kapusta, chief executive officer at uniQure. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130. We are deeply grateful for the resilience and support of the Huntington’s disease community and remain committed to standing with patients and their families as we advance this potentially transformative therapy for a community in need.”
uniQure already has an approved gene therapy for haemophilia B and is developing additional gene therapies for severe diseases.
