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Investing.com -- Rocket Pharmaceuticals Inc (NASDAQ:RCKT) stock surged 15% after the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to its gene therapy candidate for a life-threatening heart condition.
The designation was awarded to RP-A601, the company’s investigational adeno-associated virus-based gene therapy for PKP2-arrhythmogenic cardiomyopathy (ACM), a serious heart failure disease that can cause ventricular arrhythmias and sudden cardiac death. This marks the fifth RMAT designation in Rocket’s history.
RMAT designation provides significant benefits, including intensive FDA guidance and expedited review, potentially accelerating the therapy’s path to market. The FDA granted the designation based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A601.
"The FDA’s RMAT designation for RP-A601 represents a meaningful advancement for Rocket and for patients living with PKP2-ACM," said Kinnari Patel, President and Head of R&D at Rocket Pharmaceuticals.
Preliminary results from the ongoing Phase 1 trial showed encouraging early safety and efficacy data. All three adult patients treated with a single dose of RP-A601 demonstrated increased PKP2 protein expression, with two patients showing increases of 110% and 398%. Patients also experienced improvements in right ventricular function, reduced ventricular arrhythmias, and better quality of life.
The therapy was generally well-tolerated, with no dose-limiting toxicities reported and most adverse events being mild or moderate and self-limited.
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