uniQure N.V. (QURE), Thursday said U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation
to AMT-130 for the treatment of Huntingtons disease, a rare, inherited neurodegenerative disorder.
Breakthrough Therapy designation is a process to expedite the development and review of drugs that are intended to treat serious conditions where early clinical evidence indicates a substantial improvement over existing therapies.
The gene therapy candidate has already been granted Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation and Fast Track designation.
The Breakthrough Therapy designation is supported by data from the
ongoing Phase I/II studies of AMT-130 for the treatment of Huntingtons disease. In July 2024, uniQure presented interim data at 24 months that showed dose-dependent slowing of disease progression in patients treated with AMT-130.
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