uniQure’s AMT-130 Receives FDA Breakthrough Therapy Designation for Huntington’s Disease

LEXINGTON, Mass. and AMSTERDAM – April 17, 2025 – uniQure N.V. (NASDAQ: QURE), a leading gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130, an investigational gene therapy utilizing an adeno-associated virus serotype 5 (AAV5) vector, for the treatment of Huntington’s disease. This rare, inherited neurodegenerative disorder currently lacks any disease-modifying therapies. This new designation adds to the previously granted RMAT, Orphan Drug, and Fast Track designations for AMT-130.

The Breakthrough Therapy designation underscores the urgent need for effective Huntington’s disease treatments and highlights encouraging interim data suggesting AMT-130’s potential to slow disease progression, according to uniQure Chief Medical Officer Dr. Walid Abi-Saab. The designation is supported by 24-month interim data from ongoing Phase I/II trials, which showed a dose-dependent slowing of disease progression in treated patients compared to natural history data. To date, 45 patients have received AMT-130.

AMT-130 utilizes AAV5 to deliver a microRNA (miRNA) that aims to lower the production of the mutant huntingtin protein, the underlying cause of Huntington’s disease. The therapy is administered directly into the brain.

Breakthrough Therapy designation aims to accelerate the development and review of investigational therapies for serious conditions where preliminary clinical evidence indicates substantial improvement over existing options. It provides access to Fast Track features, intensive FDA guidance, and senior management involvement.

Huntington’s disease is caused by a CAG repeat expansion in the huntingtin gene, leading to abnormal protein aggregation in the brain and progressive motor, behavioral, and cognitive decline. Affecting approximately 70,000 people in the U.S. and Europe with hundreds of thousands more at risk, there are currently no approved therapies to delay the onset or to slow its progression. While there are treatments for some symptoms, AMT-130 represents a significant investigational approach targeting the disease’s root cause. uniQure is committed to working closely with the FDA to bring this potential therapy to the Huntington’s disease community as quickly as possible.