uniQure N.V. announced on April 17, 2025, that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. This designation is a critical regulatory milestone, adding to its previously granted Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations.
The Breakthrough Therapy designation is supported by clinical data from the ongoing Phase I/II trials of AMT-130. Interim data presented in July 2024 showed a dose-dependent slowing of disease progression based on the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months, compared to a propensity-weighted natural history.
This designation is intended to expedite the development and review of investigational therapies for serious conditions where preliminary clinical evidence indicates a potential for substantial improvement over available treatments. It provides uniQure with intensive guidance on an efficient drug development program and FDA commitment involving senior managers, aiming to accelerate AMT-130's path to patients.
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