Posted by Michael Wonder on 01 Jun 2023
Rocket Pharmaceuticals receives EMA Priority Medicines (PRIME) designation for RP-A501 gene therapy for Danon disease
31 May 2023 - Rocket Pharmaceuticals today announced that the EMA has granted Priority Medicines (PRIME) designation to RP-A501, the Company’s investigational adeno-associated virus 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.
