Rocket Pharmaceuticals, Inc. (RCKT) announced on Wednesday that the European Commission has granted orphan medicinal product designation for RP-A601, the company's investigational therapy for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
Currently, no curative treatments exist for PKP2-ACM, an inherited heart disorder characterized by life-threatening arrhythmias, structural cardiac abnormalities, and sudden cardiac death.
The orphan medicinal product designation by the European Commission offers several financial and regulatory advantages. These include protocol assistance from the European Medicines Agency during clinical development, access to centralized marketing authorization, and a 10-year period of market exclusivity subsequent to product approval.
