After obtaining a significant regulatory designation, investor confidence in Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) surged. Thursday saw a 16.78% increase in RCKT shares, which ended the trading day at $3.34.
The momentum comes after the U.S. Food and Drug Administration (FDA) announced that Rocket’s gene therapy candidate, RP-A601, has been designated as a Regenerative Medicine Advanced Therapy (RMAT) to treat PKP2-arrhythmogenic cardiomyopathy (ACM), a rare and potentially deadly genetic heart condition.
Designation as a Breakthrough
Positive safety and effectiveness results from RP-A601’s current Phase 1 clinical trial served as the basis for the RMAT designation. By reaching this milestone, Rocket is allowed to work with the FDA more closely, which might lead to priority evaluation of its future biologics license application (BLA), expedited development, and early input on trial design.
This is Rocket Pharmaceuticals’ seventh RMAT recognition, the company stressed, confirming its strategic commitment on creating gene treatments for hereditary cardiovascular diseases.
Promoting Outcomes Show Clinical Advantage
Notable advancements were highlighted in preliminary results given at the American Society of Gene and Cell Therapy’s 2025 Annual Meeting. When given a single dosage of RP-A601, all three adult patients showed enhanced PKP2 protein expression; in patients with originally low protein levels, this was most noticeable, with increases of 110% and 398%.
Along with favorable changes in important clinical endpoints such right ventricular function, ventricular arrhythmia reduction, and quality of life improvements, the treatment also showed improvements in desmosomal structure and function. Notably, patients’ NYHA classification improved from Class II to Class I, and their KCCQ-12 scores increased by 34–41 points.
A Positive Safety Profile Encourages Continued Improvement
There were no dose-limiting toxicities seen, and RP-A601 was generally well tolerated. The majority of unfavorable incidents were mild to moderate in intensity and went away on their own. These encouraging first results highlight Rocket’s dedication to using cutting-edge gene treatments to treat uncommon, fatal cardiac disorders and support the further development of RP-A601.
