Posted by Michael Wonder on 08 Jun 2023
Rocket Pharmaceuticals receives FDA fast track and orphan drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy
8 June 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted fast track and orphan drug designations to RP-A601, the Company’s gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
RP-A601 is Rocket’s adeno-associated virus (AAV.rh74)-based gene therapy for PKP2-ACM, a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
