08:00 AM EDT, 05/29/2024 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Wednesday that it received orphan medicinal product designation from the European Commission for RP-A601, its gene therapy for plakophilin-2-related arrhythmogenic cardiomyopathy, or PKP2-ACM.
The designation is based on the European Medicines Agency's positive opinion, the company said.
Orphan designation provides financial and regulatory benefits, including protocol assistance from the EMA during clinical development, centralized marketing authorization access, and 10 years of marketing exclusivity following product approval, the company said.
Rocket Pharmaceuticals ( RCKT ) is enrolling patients in a phase 1 trial to evaluate the safety and efficacy of RP-A601 for high-risk PKP2-ACM patients.