uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease

Core Viewpoint - uniQure N.V. is advancing its investigational gene therapy AMT-130 for Huntington's disease, with a Biologics License Application (BLA) submission planned for the first quarter of 2026, following alignment with the FDA on key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information [1][2][8] Regulatory Update - The company has engaged in productive discussions with the FDA, confirming the use of the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an acceptable clinical endpoint for accelerated approval [3][4] - The FDA has agreed to the use of external control data from the ENROLL-HD dataset, which includes approximately 33,000 patients, to enhance the robustness of the statistical analysis plan [4][5] Statistical Analysis Plan - The primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm [3] - The company plans to submit an updated statistical analysis plan to the FDA in the second quarter of 2025 [5] Chemistry, Manufacturing and Controls (CMC) - The FDA has agreed that the validation of the AMT-130 manufacturing process can leverage prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX) process [6][7] - The proposed drug product release testing plan, including the potency assay, is pending completion of qualification activities [7] Next Steps - Key upcoming milestones include submitting the updated statistical analysis plan in Q2 2025, presenting topline Phase I/II data in Q3 2025, holding a pre-BLA meeting in Q4 2025, and submitting the BLA in Q1 2026 [8][15] Clinical Program Overview - uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to assess the safety and efficacy of AMT-130 in patients with early manifest Huntington's disease [10][11] - AMT-130 has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and Breakthrough Therapy designation, marking it as the first therapy for Huntington's disease to achieve RMAT designation [11] Huntington's Disease Context - Huntington's disease is a rare neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies currently available to slow its progression [12] Company Background - uniQure is focused on gene therapy, with a pipeline that includes treatments for Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions [13]