Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A601 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.
“The FDA’s RMAT designation for RP-A601 represents a meaningful advancement for Rocket and for patients living with PKP2-ACM, a life-threatening genetic heart disease characterized by ventricular arrhythmias and sudden cardiac death,” said Kinnari Patel, PharmD, MBA, President, Head of R&D of Rocket Pharmaceuticals. “This marks the fifth RMAT designation in our history and underscores our commitment to developing potentially curative gene therapies for patients with rare and inherited cardiovascular diseases. The early clinical data for RP-A601 are highly encouraging, and we look forward to continued collaboration with the FDA throughout the program’s development.”
RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including gene therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation provides several benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).
Preliminary results from the ongoing Phase 1 clinical trial of RP-A601 presented at the 2025 Annual Meeting of the American Society of Gene and Cell Therapy demonstrated encouraging early safety and efficacy. All three adult patients treated with a single dose of RP-A601 (8×10¹³ GC/kg) showed increased PKP2 protein expression, including 110% and 398% increases in the two patients with low baseline levels, as well as improved desmosomal integrity with relocalization of key structural proteins. Improvements or stabilization were observed across clinically meaningful endpoints, including right ventricular function, ventricular arrhythmias, and quality of life highlighted by increases of 34–41 points in KCCQ-12 scores and improvements in NYHA classification from Class II to Class I. The safety profile was favorable, with RP-A601 generally well-tolerated, no dose-limiting toxicities, and most adverse events being mild or moderate and self-limited.
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