Rocket Pharmaceuticals reaches FDA alignment on Phase 2 RP-A401 trial design

Rocket Pharmaceuticals announced that alignment has been reached with the Food and Drug Administration on the global Phase 2 pivotal trial of RP-A501 for Danon Disease. Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male patients at age ~20 and females at age ~40, and for which there are no approved curative or disease-modifying therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe. “I am very excited to announce our alignment with the FDA on our pivotal study design for RP-A501 for Danon Disease, which reflects the highly collaborative discussions with the review team and senior management at FDA’s Center for Biologics Evaluation and Research and marks the first-ever regulatory pathway to approval for a genetic treatment for heart disease. We believe this milestone sets us on the most efficient and rapid path to delivering this potentially transformative therapy to Danon Disease patients who would otherwise progress to heart transplantation or death,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “I would also like to highlight the work conducted by our CMC team over the past several years to establish our in-house cGMP manufacturing capabilities, which has already provided us with sufficient material for the pivotal study and should support our eventual commercialization efforts.”