In another reversal, FDA gives uniQure accelerated approval path for Huntington's gene therapy

The FDA has reversed its earlier stance and will allow uniQure to pursue accelerated approval for its Huntington's disease gene therapy, AMT-130, based on a three‑year analysis from its Phase I/II study compared to an external control.

UniQure plans to submit a Biologics License Application (BLA) in the third quarter of 2026 for AMT‑130, avoiding the need to conduct a new Phase III trial before filing.

The FDA agreed that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) can serve as an intermediate clinical endpoint for accelerated approval, with reductions in neurofilament light chain (NfL) in cerebrospinal fluid as supportive evidence.

The agency also agreed to align on a confirmatory study design prior to BLA submission, including consideration of a concurrent control on standard‑of‑care therapy instead of a sham procedure, and expressed commitment to working expeditiously with uniQure.

AMT‑130 already holds Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Fast Track designations from the FDA, reflecting its potential to address the significant unmet need in Huntington’s disease.