The FDA has granted regenerative medicine advanced therapy designation for an investigational gene therapy to treat Huntington’s disease, according to the manufacturer.
According to a release from uniQure N.V., the FDA’s decision was based on a review of interim phase 1/2 clinical data for AMT-130 from December 2023, as well as further analysis comparing 24-month clinical data to a non-concurrent criteria-matched natural history cohort.
Regenerative medicine advanced therapy (RMAT) designation was created as part of the 21st Century Cures Act to expedite development and review