uniQure’s investigational gene therapy, AMT-130, has received Breakthrough Therapy Designation from the U.S. FDA for the treatment of Huntington’s disease, a rare, progressive neurodegenerative disorder. This designation joins earlier recognitions, including RMAT, Fast Track, and Orphan Drug status, signaling strong regulatory support for the therapy. Interim data from ongoing Phase I/II clinical trials, presented in July, showed dose-dependent slowing of disease progression compared to a matched natural history cohort. To date, 45 patients have been treated with AMT-130.
Dr. Walid Abi-Saab, uniQure’s Chief Medical Officer, emphasized the significance of the designation, noting it highlights both the unmet medical need in Huntington’s and the promise shown by AMT-130. The Breakthrough Therapy Designation is designed to expedite the development of therapies that offer substantial improvement over existing treatments.
Huntington’s disease affects around 70,000 people in the U.S. and Europe, with many more at risk. The disease causes motor dysfunction, cognitive decline, and psychiatric symptoms. Building on its success with a gene therapy for hemophilia B, uniQure is advancing a pipeline that also targets temporal lobe epilepsy, Fabry disease, and ALS, reinforcing its position as a leader in genomic medicine.
Source:
https://www.pharmaceutical-technology.com/news/fda-uniqure-huntingtons/
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