Rocket Pharmaceuticals announced alignment with the FDA on key elements for the global pivotal Phase 2 trial of RP-A501 for Danon disease. The FDA confirmed a recalibrated dose of 3.8 × 10¹³ genome copies per kilogram and a 12-patient pivotal population, including the first three patients already treated under the modified protocol. Nine additional patients remain to be enrolled, with dosing expected to complete by mid-2027. The trial utilizes established 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index to support potential accelerated approval. Initial safety data from the first three patients showed no evidence of thrombotic microangiopathy or capillary leak syndrome. Rocket Pharmaceuticals is a commercial-stage biotechnology company developing genetic medicines for rare and life-threatening diseases, with a strategic focus on inherited cardiovascular disorders.
