Rocket Pharma’s RP-A601 Gene Therapy Granted FDA RMAT Designation for PKP2-ACM

Rocket Pharmaceuticals, Inc. 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 gene therapy for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a rare and potentially fatal genetic heart condition.

The RMAT designation, which is intended to accelerate the development of regenerative medicine therapies for serious or life-threatening conditions, was awarded based on positive early results from Rocket’s ongoing Phase 1 trial. The designation will enable more intensive FDA guidance and an expedited review process, including potential priority review of a future biologics license application (BLA).

PKP2-ACM is a severe inherited disease marked by life-threatening ventricular arrhythmias and sudden cardiac death. RP-A601, an adeno-associated virus (AAV)-based gene therapy, aims to correct the underlying genetic defect. Preliminary Phase 1 data presented at the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting showed promising results: all three adult patients treated with a single dose exhibited increased PKP2 protein expression, improved heart structure and function, and notable enhancements in quality of life. No dose-limiting toxicities were observed.

“This marks our fifth RMAT designation and reinforces our mission to deliver potentially curative gene therapies to patients with rare cardiovascular diseases,” said Kinnari Patel, PharmD, MBA, President and Head of R&D at Rocket. “We are encouraged by the early clinical data for RP-A601 and look forward to working closely with the FDA as we advance development.”

The RMAT program, established under the 21st Century Cures Act, is designed to speed up the review of transformative therapies, offering benefits such as early dialogue with the FDA and flexibility around endpoints that could support accelerated approval.