FDA no longer considers AMT-130 Phase I/II vs external control adequate for BLA

uniQure N.V.

Pre-BLA meeting feedback: FDA believes data from Phase I/II of AMT-130 vs external control may not serve as primary evidence for BLA.
This reverses prior FDA guidance from multiple Type B meetings over past year and Breakthrough Therapy designation in April 2025.
BLA submission timing for AMT-130 in Huntington's disease is now unclear.
Company expects final meeting minutes within 30 days and plans to urgently engage FDA.
uniQure also plans to pursue parallel discussions with EU and UK regulators.

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