uniQure plans Q3 2026 BLA filing for AMT-130 after FDA agrees 3-year data can support submission

Filed June 17, 2026 · Period ending June 17, 2026 · ~1 min read

Key Changes

• high

  FDA agreed in Type B meeting that 3-year Phase I/II data would be acceptable as primary basis for accelerated approval BLA for AMT-130 in Huntington's disease

  Item 8.01 verify on EDGAR →
• high

  Company intends to submit BLA in Q3 2026, contingent on aligning with FDA on confirmatory study design prior to submission

  Item 8.01 verify on EDGAR →
• high

  FDA seeks alignment on confirmatory study design before BLA submission, considering concurrent standard-of-care control instead of sham procedure

  Item 8.01 verify on EDGAR →