uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington's Disease

Core Insights - The U.S. FDA has granted Breakthrough Therapy designation to AMT-130 for Huntington's disease, highlighting the urgent need for effective treatments and the promising interim data indicating potential disease progression slowing [1][2] - The Breakthrough Therapy designation is in addition to other FDA designations previously granted to AMT-130, including RMAT, Orphan Drug, and Fast Track [1] - The company anticipates additional regulatory updates and guidance on the Biologics License Application submission in the second quarter of 2025 [1] Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs, including Huntington's disease, ALS, and Fabry disease [5] - The company has made significant progress in gene therapy, evidenced by the approval of its gene therapy for hemophilia B, marking a milestone in genomic medicine [5] Huntington's Disease Context - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies available to slow its progression [4] - The disease is characterized by motor symptoms, behavioral abnormalities, and cognitive decline due to a genetic mutation in the huntingtin gene [4] Clinical Data and Development - Interim data from ongoing Phase I/II trials of AMT-130 presented in July 2024 showed dose-dependent slowing of disease progression based on the cUHDRS scale, with 45 patients treated to date [2][3] - The Breakthrough Therapy designation aims to expedite the development and review of investigational therapies that show substantial improvement over existing treatments [3]