FDA Grants Fast Track Designation to Rocket’s Gene Therapy for Rare Heart Condition

Rare Daily Staff

The U.S. Food and Drug Administration granted Rocket Pharmaceuticals Fast Track and Orphan Drug designations to RP-A601, the company’s experimental gene therapy for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy, a rare heart condition.

PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. Patients living with PKP2-ACM are treated with implantable cardioverter defibrillators and ablation therapies, but these do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation.

RP-A601 is being developed as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by this devastating disease. It is an adeno-associated virus-based gene therapy for PKP2-ACM. The current standard of care consists of medical therapy, implantable cardioverter defibrillators, and ablation procedures, none of which are curative. Even with treatment, life-threatening arrhythmias and progression of disease can still occur.

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 application review.

Orphan Drug designation is granted to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States. The designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.