uniQure N.V. provided a regulatory update on June 2, 2025, for AMT-130, its investigational gene therapy for Huntington’s disease, confirming continued alignment with the U.S. Food and Drug Administration (FDA) on key elements for an Accelerated Approval pathway. The company plans to submit a Biologics License Application (BLA) in the first quarter of 2026.
Following recent Type B meetings, the FDA agreed on the proposed use of external control data and the prospectively defined statistical analysis plan (SAP). The primary efficacy analysis for the BLA will evaluate the 3-year change in composite Unified Huntington’s Disease Rating Scale (cUHDRS) in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm derived from the ENROLL-HD dataset.
Regarding Chemistry, Manufacturing and Controls (CMC) requirements, the FDA agreed that validation of the AMT-130 manufacturing process can leverage experience from the HEMGENIX process, supplemented by additional full-scale GMP batches and a single Process Performance Qualification (PPQ) batch. The FDA also agreed with the proposed drug product release testing plan, pending completion of qualification and specification setting activities. These agreements provide a clear path forward for the BLA submission.
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