uniQure reaches FDA alignment on AMT-130 BLA for Huntington's disease; submission by Q1 2026

uniQure N.V.

FDA supports cUHDRS as accelerated approval endpoint; primary analysis uses 3-year cUHDRS change vs propensity score-weighted ENROLL-HD external control.
CMC validation: FDA agrees to use prior HEMGENIX knowledge, plus additional AMT-130 GMP batches and one PPQ batch; potency assay plan accepted pending qualification.
BLA submission targeting Q1 2026 with request for priority review; pre-BLA meeting in Q4 2025; Phase I/II topline data in Q3 2025.
Type B meetings held in Q1 and Q2 2025 on CMC and SAP; FDA aligned on statistical analysis plan including sensitivity analyses using TRACK-HD and PREDICT-HD datasets.

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