Rocket Pharmaceuticals Gets European Medicines Agency (EMA) Priority Medicines (PRIME) Designation For RP-A501 Gene Therapy For Danon Disease
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Rocket Pharmaceuticals has been granted PRIME designation by the European Medicines Agency (EMA) for its investigational gene therapy RP-A501 for the treatment of Danon Disease. The designation is based on positive safety and efficacy data from a Phase 1 clinical trial.

May 31, 2023 | 11:11 am
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Rocket Pharmaceuticals' RP-A501 gene therapy for Danon Disease has received PRIME designation from the EMA, potentially accelerating its development and approval process.
The PRIME designation for Rocket Pharmaceuticals' RP-A501 gene therapy is a significant milestone, as it indicates positive safety and efficacy data from the Phase 1 clinical trial. This designation could accelerate the development and approval process for the therapy, potentially leading to increased revenues and a positive impact on the company's stock price in the short term.
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