FDA Reverses on uniQure's Huntington's Disease Gene Therapy, Clearing Q3 2026 BLA Path
On June 17, 2026, uniQure announced that the FDA has performed a major regulatory U-turn, agreeing that the 3-year clinical analysis from the Phase I/II study of AMT-130 is acceptable as the primary evidentiary basis for a Biologics License Application (BLA) under the accelerated approval pathway. This represents a significant reversal from the FDA's previous stance in March 2026, which had rejected using this early data for a filing. uniQure now plans to formally submit the BLA in the third quarter of 2026.
AMT-130 is an investigational gene therapy designed to inhibit the production of mutant huntingtin protein in patients with early manifest Huntington’s disease. It is administered via a single, MRI-guided stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).
Regulatory Alignment and Confirmatory Trial Design
A crucial aspect of the FDA's new position is the commitment to work "as expeditiously as possible" to align on the design of the post-approval confirmatory study before the BLA is submitted. Notably, the FDA is open to considering a concurrent standard-of-care control arm rather than requiring a sham neurosurgical procedure (imitation surgery), which has been a major ethical and logistical hurdle for clinical trials in this space.
This shift in flexibility at the FDA is viewed by industry analysts as part of a broader trend of regulatory softening following leadership changes at the agency, including the departures of officials who had favored stricter standards for accelerated approvals.1
"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease. The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study." — Matt Kapusta, Chief Executive Officer of uniQure
-
An instance of Leadership churn at regulatory agencies triggers a systemic softening of clinical standards. — The exit of conservative FDA leadership enabled a more flexible regulatory environment that permitted uniQure to seek approval based on earlier Phase I/II trials. ↩︎