uniQure to submit BLA for AMT-130 in Huntington's disease in Q3 2026 after FDA Type B meeting

uniQure N.V.

• FDA agreed 3-year Phase I/II analysis can serve as primary basis for accelerated approval BLA for AMT-130.
• Company plans BLA submission in Q3 2026; FDA seeks alignment on confirmatory study with standard-of-care control.
• AMT-130 has RMAT, Breakthrough Therapy, and Fast Track designations from FDA.
• 3-year data from 26 US and 13 EU patients compared against propensity-matched external control Enroll-HD dataset.