Pharma Focus Asia

Rocket Pharmaceuticals Announces FDA Clearance of Investigational New Drug (IND) Application for RP-A601

Tuesday, May 09, 2023

Rocket Pharmaceutical announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy. 

RP-A601 offers the potential of a one-time, curative alternative to medical therapy, ICDs, and ablations which are associated with adverse effects, complications, and recurrence of arrhythmias and do not halt the progression of disease.

The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative. Even with treatment, life-threatening arrhythmias and progression of disease may still occur. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe.
PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterised by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that will improve survival and quality of life for patients affected by this devastating disease.

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