uniQure plans Q3 2026 BLA submission for AMT-130 gene therapy after FDA greenlights accelerated approval pathway

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

Key Changes

• high

  FDA confirmed 3-year Phase I/II data sufficient for accelerated approval BLA submission of AMT-130, a gene therapy for Huntington's Disease, planned for Q3 2026

  Item 8.01 verify on EDGAR →
• high

  Accelerated approval pathway could bring AMT-130 to market faster than traditional approval for this serious condition with limited treatment options

  8-K: FDA Meeting verify on EDGAR →
• medium

  FDA requires agreement on confirmatory study design before BLA submission, considering standard-of-care control instead of sham procedure

  Item 8.01 verify on EDGAR →