uniQure reaches FDA alignment on Accelerated Approval pathway for AMT-130 in Huntington's disease

uniQure N.V.

FDA agrees data from ongoing Phase I/II studies vs natural history external control may serve as primary basis for BLA under Accelerated Approval.
FDA agrees cUHDRS may serve as intermediate clinical endpoint; reductions in CSF NfL provide supportive evidence of benefit.
Company initiated BLA readiness activities; plans to discuss statistical analysis plan and CMC requirements with FDA in H1 2025.

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