Gene Therapy Gets Fast Track, Orphan Drug Status for PKP2 Arrhythmogenic Cardiomyopathy

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RP-A601 is an investigational adeno-associated virus-based gene therapy.

The Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), an inherited heart disease caused by mutations in the PKP2 gene.

Individuals with PKP2-ACM have been known to experience life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. RP-A601 is an adeno-associated virus (AAV.rh74)-based gene therapy that is being investigated as a one-time curative treatment for these patients. The FDA’s designations are supported by preclinical studies indicating that the gene therapy candidate has the potential to decrease arrhythmias and increase survival.

An upcoming phase 1 dose escalation trial will evaluate the safety and efficacy of RP-A601 in at least 6 high-risk adult patients with PKP2-ACM ( Identifier: NCT05885412). According to Rocket Pharmaceuticals, the study will assess the impact of the gene therapy on PKP2 myocardial protein expression, cardiac biomarkers, clinical predictors of life-threatening ventricular arrhythmias, and sudden cardiac death.

Approximately 50,000 adults and children in the US and Europe are affected by PKP2-ACM. The current standard of care includes medical therapy, implantable cardioverter defibrillators, and ablation procedures.


Rocket Pharmaceuticals receives FDA Fast Track and Orphan Drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy (ACM). News release. June 8, 2023.