Rocket Pharmaceuticals Gets Orphan Drug Status from European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a biotechnology company based in Cranbury, N.J., has announced that the European Commission (EC) has granted orphan medicinal product designation for their gene therapy candidate RP-A601. This decision was influenced by a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). RP-A601 is an innovative gene therapy designed to treat plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), a severe inherited heart disease.

PKP2-ACM is characterized by life-threatening arrhythmias, structural heart abnormalities, and sudden cardiac death, impacting approximately 50,000 people in the U.S. and Europe. Currently, there are no curative treatments available for PKP2-ACM. Standard care includes medical therapy, implantable cardioverter defibrillators (ICDs), and ablation procedures, but these treatments do not prevent disease progression and can still result in life-threatening arrhythmias.

The orphan medicinal product designation by the EC applies to therapies designed to treat or prevent life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU). This designation provides financial and regulatory benefits, including protocol assistance from the EMA during clinical development, centralized marketing authorization, and a 10-year period of marketing exclusivity post-approval.

Rocket Pharmaceuticals is currently enrolling patients in a Phase 1, dose-escalation trial to assess the safety and initial efficacy of RP-A601. This trial involves at least six adult PKP2-ACM patients with ICDs, who are at high risk for severe arrhythmias. The study aims to evaluate the impact of RP-A601 on myocardial protein expression, cardiac biomarkers, clinical indicators of life-threatening ventricular arrhythmias, and sudden cardiac death.

RP-A601 is an experimental gene therapy that utilizes a recombinant adeno-associated serotype rh74 (AAVrh74) capsid. This capsid contains a functional version of the human PKP2 transgene (AAVrh74.PKP2), administered as a single intravenous infusion. The therapy aims to be a one-time, potentially curative treatment that could significantly improve survival and quality of life for PKP2-ACM patients. Rocket Pharmaceuticals holds Fast Track designation in the U.S. and Orphan Drug designation in both the U.S. and Europe for RP-A601.

PKP2-ACM is caused by mutations in the PKP2 gene, leading to severe ventricular arrhythmias, structural heart defects, and sudden cardiac death. The current medical approach, including ICDs and ablation procedures, does not consistently prevent disease progression or recurrence of arrhythmias and is associated with significant morbidity. There is a critical need for treatments that address the underlying genetic mutations and pathophysiology of the disease.

Rocket Pharmaceuticals is a late-stage biotechnology company focusing on genetic therapies for rare and complex disorders. The company employs a multi-platform approach to develop transformative gene therapies. Its portfolio includes advanced programs for conditions like Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD), and cardiovascular diseases such as Danon Disease and PKP2-ACM.

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