On February 7, 2023, Rocket Pharmaceuticals Inc., a renowned late-stage biotech firm advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet needs, announced that FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease, a life-threatening genetic cardiac disease with no disease-altering therapies available. RMAT status was granted based on favorable safety and effectiveness findings from the Phase 1 RP-A501 clinical trial and will provide further added benefits of intensive FDA guidance and accelerated evaluation throughout the program's development.
RMAT designation, the same as Breakthrough Therapy designation, offers the advantages of intensive FDA guidance on efficient drug development, such as the ability for early FDA interactions to discuss intermediate or surrogate endpoints, potential priority review of the Biologics License Application (BLA), possible ways to support accelerated approval and satisfy post-approval requirements, and other possibilities to expedite development and assessment. Rocket's RP-A501 program also has Orphan Drug (US) and Rare Pediatric designations.
The Phase 2 pivotal trial is scheduled to begin in the second quarter of 2023. As disclosed previously, the company plans to conduct a single-arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.
The Phase 1 program's findings provide one of the most extensive investigational gene therapy datasets for any cardiac disease. In patients with Danon Disease treated in the Phase 1 study, RP-A501 was generally well tolerated, with indications of restored expression of the deficient LAMP2 protein and durable improvement or stability of clinical parameters.
Data show consistent and robust improvements in a variety of parameters, including protein expression, decreased autophagic vacuoles, troponin, brain natriuretic peptide (BNP), left ventricular (LV) mass and thickness, and enhanced NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. Notably, these BNP and NYHA class improvements and stability contrast sharply with BNP rise and NYHA class worsening found in a representative sample of pediatric and adolescent natural history patients. Overall, the results indicate improvements and normalization in a variety of quantifiable parameters used by cardiologists in clinical practice to foster risk assessment and treatment decisions.
About Rocket Pharmaceuticals, Inc
Founded in 2015, Rocket Pharmaceuticals is a fully integrated clinical-stage firm developing an integrated and sustainable pipeline of investigational genetic therapeutics that have the potential to address the underlying causes of complex rare childhood diseases. Through its platform-agnostic approach, it can build the optimal therapy for each indication, possibly generating revolutionary options for patients suffering from rare genetic disorders. The company has a team of highly productive experts and is increasingly being recognized as a leading biotechnology innovator.