The gene therapy is currently under investigation as a one-time curative treatment. Credit: R Photography Background / Shutterstock.com.

The European Commission (EC) has granted orphan medicinal product designation for Rocket Pharmaceuticals’ RP-A601 gene therapy candidate to treat plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).

PKP2-ACM is an inherited heart disease that poses a significant risk of arrhythmias, cardiac abnormalities and sudden cardiac death.

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The decision follows a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).

RP-A601 is a recombinant adeno-associated serotype rh74 (AAVrh74) capsid containing a functional version of the human PKP2 transgene.

It is administered as a single intravenous infusion and is currently under investigation as a one-time, potentially curative treatment.

The therapy aims to improve survival and quality of life for patients with PKP2-ACM, a condition for which there are no curative treatments available.

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An orphan medicinal product designation is awarded to new therapies for rare, life-threatening or chronically debilitating conditions.

In the European Union, this applies to diseases affecting fewer than five in 10,000 persons. Benefits of the designation include protocol assistance from the EMA, access to centralised marketing authorisation and a decade of marketing exclusivity post-approval.

RP-A601 has also obtained fast-track and orphan drug designations in the US.

Rocket Pharmaceuticals is enrolling patients in a Phase I dose escalation trial to evaluate RP-A601’s safety and initial efficacy.

The trial aims to include a minimum of six adult patients with PKP2-ACM who are at high risk for life-threatening arrhythmias and have implantable cardioverter defibrillators.

It will assess RP-A601’s impact on PKP2 myocardial protein expression, cardiac biomarkers and clinical predictors of ventricular arrhythmias and sudden cardiac death.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.