uniQure Plans Q3 2026 BLA Submission for AMT-130 Based on 3-Year Data
QURE•uniQure received FDA confirmation that its 3-year Phase I/II analysis in 26 early-manifest Huntington’s disease patients will serve as the primary basis for an accelerated BLA, with submission slated for Q3 2026. FDA also requested alignment on the confirmatory study design, including a concurrent standard-of-care control arm.
1. FDA Accepts 3-Year Phase I/II Data for BLA
The FDA agreed that uniQure’s 3-year analysis of its Phase I/II study in Huntington’s disease—enrolling 26 early-manifest patients across low- and high-dose cohorts with propensity-score matched external controls—can serve as the primary basis for an accelerated Biologics License Application.
2. Planned Q3 2026 BLA Submission
uniQure intends to submit the BLA for AMT-130 in the third quarter of 2026 and anticipates receiving final meeting minutes within 30 days of the recent Type B meeting, advancing the therapy toward potential accelerated approval.
3. Confirmatory Study Design Alignment
The FDA requested that uniQure align on the confirmatory trial design prior to BLA submission, favoring a concurrent standard-of-care control arm over a sham procedure, and committed to work expeditiously on study parameters.
4. Regulatory Designations and Pipeline Impact
AMT-130 has secured Regenerative Medicine Advanced Therapy, Breakthrough Therapy and Fast Track designations, reinforcing its priority status and complementing uniQure’s expanding gene therapy pipeline following hemophilia B approval.
