FDA grants regenerative medicine advanced therapy designation for Huntington’s treatment

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 processes of regenerative medicine therapies. A regenerative medicine therapy can be eligible for RMAT designation if it is developed to treat, modify, reverse or cure a serious condition and shows preliminary clinical evidence that the drug is likely to address unmet medical needs.

According to the release, interim data from the ongoing phase 1/2 United States and European studies of AMT-130 will be released this year. The data is expected to include up to 3 years of follow-up on 29 treated participants, 21 of whom will have been followed for at least 2 years.

“We’re thrilled to receive the first ever RMAT designation for an investigational therapy for Huntington’s disease,” uniQure CEO Matt Kapusta said in the release. “This achievement is a significant milestone for the program and supports the potential for AMT-130 to address the high unmet medical need of those suffering from this devastating disease.”