Rocket Pharmaceuticals announced that the FDA has granted Regenerative Medicine Advanced Therapy, or RMAT, designation to RP-A501, the company’s investigational adeno-associated virus, or 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.
Published first on TheFly
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