FDA Grants RMAT Status to UniQure's AMT-130 Gene Therapy for Huntington's Disease

Benzinga · 06/03 11:17

uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) 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.