Rocket Pharmaceuticals Receives FDA Fast Track and Orphan Drug Designations for RP-A601 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced 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. The current standard of care consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablation procedures, none of which are curative. Even with treatment, life-threatening arrhythmias and progression of disease can still occur. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe.

Fast Track designation is granted to facilitate the development and expedite the review of medicines that treat serious conditions and fill an unmet medical need. The designation enables increased communication with the FDA throughout the development of RP-A601, offers the potential for accelerated approval and priority review if criteria are met, and permits a Rolling BLA Review. Orphan Drug designation is granted to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the U.S.

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. RP-A601 is the first gene therapy in development for PKP2-ACM to receive IND clearance and the second of three programs in the Company’s AAV-based cardiovascular gene therapy franchise.

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. The study will assess the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, clinical predictors of life-threatening ventricular arrhythmias, and sudden cardiac death.