Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A501 Gene Therapy for Danon Disease

Feb 07, 2023

Rocket+Pharmaceuticals%2C+Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A501 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.

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