Clinical Trial SuccessThe data from the first three patients in the Ph1 study of RP-A601 demonstrated a consistent and significant decrease in arrhythmia burden, and stabilization or improvement in both right ventricular function and patients' quality of life from RP-A601 treatment.
FDA Approval PathwayAn agreement with the FDA on an accelerated approval pathway for RP-A601 is seen as possible based on a favorable clinical profile and the precedent of RP-A501 for Danon disease.
Regulatory ProgressThe FDA lifted the clinical hold on the pivotal Ph2 study of RP-A501 in Danon disease, removing a major hurdle for Rocket Pharma.