Rocket Pharma Says FDA Grants Fast Track And Orphan Drug Designations For RP-A601 Gene Therapy
(RTTNews) - Biotechnology company Rocket Pharmaceuticals, Inc. (RCKT) announced Thursday that the U.S. Food and Drug Administration (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.
Rocket recently announced FDA clearance of the Investigational New Drug (IND) application for RP-A601 based on robust preclinical proof of concept studies that demonstrated decreased arrhythmias and increased survival.
The Company is initiating a multi-center Phase 1, dose escalation trial that will evaluate the safety and preliminary efficacy of RP-A601 in at least six adult PKP2-ACM patients with ICDs and who have overall high risk for life-threatening arrhythmias.
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