Rocket Pharmaceuticals' Gene Therapy Trial for Danon Disease Faces FDA Hold After Patient Death
Rocket Pharmaceuticals announced an update on its Phase 2 trial of RP-A501 for Danon disease after a patient experienced a serious adverse event and subsequently passed away, leading to a clinical hold by the FDA. The company is investigating the cause, working with regulators and stakeholders, and has paused dosing to ensure patient safety. The trial aims to evaluate the efficacy of RP-A501, a gene therapy targeting cardiac function in Danon disease, with ongoing efforts to resume the study.