Rocket Pharmaceuticals (NASDAQ:RCKT) shares jumped more than 40% intra-day today after the company announced an agreement with the U.S. FDA regarding the Phase 2 trial of RP-A501.
This trial targets Danon disease, a life-threatening inherited cardiomyopathy. The CEO of Rocket Pharmaceuticals, Gaurav Shah, expressed confidence that this agreement represents a significant step toward efficiently and swiftly providing potential therapy to Danon Disease patients, potentially avoiding the need for heart transplantation or the risk of death.