uniQure receives RMAT designation of AMT-130 in Huntington’s Disease

uniQure announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation for its investigational gene therapy AMT-130 for the treatment of Huntington’s disease. The FDA granted RMAT designation based on the potential of AMT-130 to address the major unmet medical need among patients with Huntington’s disease. The designation follows the FDA’s review of interim Phase I/II clinical data for AMT-130 announced in December 2023 and is based on an analysis comparing these 24-month clinical data to a non-concurrent criteria-matched natural history cohort. uniQure expects to provide updated interim data from the ongoing Phase I/II U.S. and European studies of AMT-130 in the middle of 2024. The interim update will include up to three years of follow-up on 29 treated patients, 21 of which will have been followed for at least two years.