uniQure’s AMT-130 Snags FDA Breakthrough Therapy Nod for Huntington’s

April 17, 2025

DENVER, Colo., Apr 17, 2025 (247marketnews.com)- uniQure (NASDAQ:QURE) scored a monumental FDA Breakthrough Therapy designation for its AMT-130 in treating Huntington’s disease, a rare, devastating neurodegenerative disorder with no disease-modifying therapies. The designation, backed by Phase I/II trial data showing dose-dependent slowing of disease progression, catapults QURE into an estimated $10 billion neurodegenerative market.

AMT-130, uniQure’s pioneering gene therapy, tackles Huntington’s, a brutal autosomal dominant condition driven by a CAG repeat in the huntingtin gene, afflicting 70,000 diagnosed patients in the U.S. and Europe, with hundreds of thousands at risk. Interim data from July 2024, covering 45 treated patients over 24 months, revealed dose-dependent slowing of disease progression via the cUHDRS scale compared to a propensity-weighted natural history cohort.

Walid Abi-Saab, M.D., uniQure’s chief medical officer, commented, “Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression. It’s a powerful recognition of the promise of AMT-130 and the important progress we’ve made. We deeply value the FDA’s continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington’s disease patient community as quickly as possible.”

The Breakthrough Therapy nod, granted for therapies showing substantial improvement over existing options, underscores AMT-130’s edge, as no approved treatments currently slow Huntington’s relentless motor, behavioral, and cognitive decline.

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