AMT-130

June 17, 2026: uniQure N.V. (NASDAQ: QURE) said that in a recent FDA Type B meeting, the agency indicated that the 3-year analysis from the Phase I/II program for AMT-130 in Huntington’s disease would be acceptable as the primary basis for a Biologics License Application (BLA) seeking accelerated approval. The FDA also wants alignment on the confirmatory study design before the BLA is submitted, including considering a concurrent standard-of-care control arm rather than a sham procedure. uniQure said it plans to conduct the confirmatory study without delay and expects to align with FDA on details prior to submission; final meeting minutes are expected within 30 days. The company intends to submit the BLA in 3Q 2026.

AMT-130 has FDA RMAT (noted as the first in Huntington’s disease), Breakthrough Therapy, and Fast Track designations. The Phase I/II program includes U.S. and European studies in early manifest Huntington’s disease, with cohorts 1 and 2 to be compared to a propensity score–matched external control from the Enroll-HD dataset under a prespecified statistical analysis plan. Clinical trials listed: NCT05243017 and NCT04120493.