LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- In a significant advancement for the treatment of Huntingtons disease, uniQure N.V. (NASDAQ: QURE), a pioneering gene therapy firm, has announced that the U.S. Food and Drug Administration (FDA) has officially granted Breakthrough Therapy designation to its investigational drug, AMT-130. This rare, inherited neurodegenerative disorder currently lacks any disease-modifying therapies, making this milestone particularly notable for patients and their families.

Huntingtons disease is a relentless condition that leads to severe motor and cognitive decline, fundamentally altering the lives of those affected. The Breakthrough Therapy designation recognizes the urgent need for effective treatments and acknowledges the promising interim clinical data that suggests AMT-130 has the potential to significantly slow disease progression.

Dr. Walid Abi-Saab, the chief medical officer of uniQure, expressed, "Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntingtons disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression. Its a powerful recognition of the promise of AMT-130 and the important progress weve made. We deeply value the FDAs 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 Huntingtons disease patient community as quickly as possible."

The Breakthrough Therapy designation is an initiative by the FDA designed to expedite the development and review process for therapeutic candidates that aim to treat serious conditions. This status is granted when preliminary clinical evidence indicates that a drug may demonstrate substantial improvement over existing therapies on clinically significant endpoints. AMT-130's designation is complemented by several other recognitions from the FDA, including Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, and Fast Track designation, all of which streamline the regulatory process for innovative treatments.

Clinical data supporting this designation comes from ongoing Phase I/II trials of AMT-130, which have showcased interim results that indicate a dose-dependent slowing of disease progression in treated patients. In July 2024, uniQure presented interim data at the 24-month mark, illustrating these promising effects. To date, 45 patients have participated in the AMT-130 trials, which aim to provide a new hope for those battling Huntingtons disease.

Huntingtons disease is caused by a genetic mutation leading to the accumulation of toxic protein in the brain, primarily affecting motor function, behavior, and cognitive abilities. According to a 2021 study published in Neuroepidemiology, approximately 70,000 individuals are diagnosed with Huntingtons disease across the U.S. and Europe, with many more at risk due to familial inheritance. Currently, there are no approved therapies capable of delaying the onset or progression of this devastating disorder.

uniQure is at the forefront of gene therapy, with a commitment to delivering potentially curative treatments through single-dose interventions. The company has already made history with its gene therapy for hemophilia B, and it is now focusing on developing a pipeline of proprietary gene therapies targeting conditions such as Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease. The organization is dedicated to transforming the landscape of treatment options for patients suffering from severe illnesses.

As part of its forward-looking statements, uniQure emphasizes the importance of regulatory interactions and clinical trial outcomes, which are critical for the progression of AMT-130 through approval processes. The company is keen on further communications with the FDA to refine its Biologics License Application (BLA) submission, anticipated in the second quarter of 2025. They are also preparing to share more interim data and updates from ongoing trials in the near future.