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 European Medicines Agency (EMA) has granted Priority Medicines (PRIME) 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 inherited cardiomyopathy for which there are no curative therapies. PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease and the potential of RP-A501 to meet the high unmet medical need in this population.
Rocket Pharmaceuticals Receives European Medicines Agency (EMA) Priority Medicines (PRIME) Designation for RP-A501 Gene Therapy for Danon Disease
May 31, 2023
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