GlobeNewswire·Jun 17, 2026, 7:05 AM ET

uniQure Announces Plan for BLA Submission for AMT-130 in Huntington's Disease

The FDA accepted the 3-year analysis from the Phase I/II study as the primary basis for a Biologics License Application (BLA) for the accelerated approval of AMT-130 for Huntington's disease.
uniQure intends to submit the BLA in the third quarter of 2026.
The FDA seeks to align on a confirmatory study design that utilizes a concurrent standard-of-care control instead of a sham procedure.
The FDA committed to working expeditiously with uniQure to finalize the confirmatory study details prior to the BLA submission.
The company expects to receive the final minutes of the recent Type B meeting within 30 days.
AMT-130 holds Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, & Fast Track designations from the FDA.
The Phase I/II clinical program includes multiple cohorts evaluating safety, tolerability, & efficacy in patients with early manifest Huntington's disease using MRI-guided delivery into the striatum.
FDA discussions confirmed that data from cohorts 1 & 2 could be compared to a propensity score-matched external control derived from the Enroll-HD natural history dataset.
Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 75,000 people in the U.S., EU, & UK, with no currently approved therapies to slow progression.