RCKT Stock Up on FDA's RMAT Tag to Gene Therapy for Heart Failure

Core Insights - Rocket Pharmaceuticals (RCKT) has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for its gene therapy candidate RP-A601, aimed at treating PKP2-arrhythmogenic cardiomyopathy (ACM) [1][7] - The RMAT designation allows for enhanced interactions with the FDA, facilitating accelerated approval processes and early discussions on potential endpoints [2] Company Developments - Following the RMAT announcement, RCKT's shares increased by 16.8%, although the stock has decreased by 73.5% year-to-date, contrasting with a 0.7% decline in the industry [3] - The FDA's RMAT designation was based on positive safety and efficacy data from a phase I study, which indicated that RP-A601 is generally safe and well-tolerated [4][7] - RCKT is also developing another gene therapy candidate, RP-A701, for BAG3-associated dilated cardiomyopathy (BAG3-DCM), with the FDA recently approving its investigational new drug application [5][8] Recent Challenges - The company faced setbacks, including a voluntary pause in the pivotal phase II study of RP-A501 for Danon disease due to a patient death, leading to an FDA clinical hold [9] - Additionally, the FDA issued a complete response letter regarding the BLA for Kresladi, requesting more information on Chemistry Manufacturing and Controls, which has delayed the review process [10]