Gene therapy for inherited cardiomyopathy gains FDA fast track, orphan drug designation
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Rocket Pharmaceuticals announced that the FDA has granted fast track and orphan drug designations to its adeno-associated virus-based gene therapy for the treatment of plakophilin-2-related arrhythmogenic cardiomyopathy.
The gene therapy (RP-A601) is the first to receive investigational new drug clearance for the treatment of plakophilin-2-related arrhythmogenic cardiomyopathy, according to a company press release.
The gene therapy is planned for testing in a multicenter phase 1 dose-escalation trial to evaluate its safety and preliminary efficacy in at least six high-risk adult patients with plakophilin-2-related arrhythmogenic cardiomyopathy and implantable cardioverter defibrillators, according to the release.
The fast track designation is granted to therapies with the potential to fill unmet needs of patients with serious medical conditions. It includes the ability for the developer to have more frequent interactions with the agency, and potentially future accelerated priority review and accelerated approval. Orphan drug designation is granted for the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases with a prevalence of less than 200,000 patients annually.
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