Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug Administration (FDA), which granted its investigational gene therapy RP-A601 the Regenerative Medicine Advanced Therapy (RMAT) designation.
The therapy is being developed to treat PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a rare and potentially fatal inherited heart condition associated with arrhythmias and sudden cardiac death. RP-A601 uses an adeno-associated virus (AAV) vector to deliver a corrective version of the PKP2 gene.
This is the fifth RMAT designation for Rocket Pharmaceuticals. The status provides benefits such as enhanced interaction with the FDA, priority review, and the potential for accelerated approval pathways—significantly speeding the therapy’s route to market.
“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.
The FDA’s decision was based on early data from a Phase 1 clinical trial, which showed promising safety and efficacy. All three adult participants in the study received a single dose of RP-A601. Two of the three showed PKP2 protein expression increases of 110% and 398%, along with improvements in heart function, fewer arrhythmias, and enhanced quality of life.
The treatment was generally well-tolerated, with no dose-limiting toxicities observed. Reported adverse events were mostly mild or moderate and resolved on their own.
Rocket Pharmaceuticals said it will continue advancing RP-A601 through clinical development as part of its broader gene therapy pipeline.
Rocket Pharmaceuticals stock price
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