Enrollment CompletionRocket Pharma announced the completion of enrollment for its Phase II pivotal study with RP-A501 in Danon disease, which highlights strong demand for the treatment.
Positive Study DataData from the Phase I study of RP-A501 de-risks the pivotal trial, as significant improvements in both co-primary endpoints were seen across all six treated patients.
Potential Accelerated ApprovalExamining RP-A501’s registrational study design shows meaningful potential for an accelerated approval, given the consistent outcomes observed across patients.