Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal inherited cardiomyopathy for which there are no curative therapies. PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease and the potential of RP-A501 to meet the high unmet medical need in this population.
PRIME designation offers the benefits of early and enhanced support from the EMA for the development of medicines that target unmet medical needs, as well as the opportunity for an accelerated review of the marketing application. Rocket was also recently granted Regenerative Medicine Advanced Therapy (RMAT) designation for its RP-A501 program, which also holds Fast Track, Orphan Drug (U.S.) and Rare Pediatric designations.
“PRIME designation from the EMA further highlights the positive benefit/risk profile of RP-A501 in addressing the critical unmet need of patients facing Danon Disease,” said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma. “We are thrilled by the opportunity that PRIME grants us, so that we may collaborate with our European Regulatory partners on the development of RP-A501 in the most expedient and efficient path forward.”
Results from the Phase 1 trial represent one of the most comprehensive investigational gene therapy datasets for any cardiac condition. RP-A501 was associated with a favorable safety profile. The data demonstrated consistent and robust improvements in multiple clinical and highly relevant laboratory parameters including LAMP-2 protein expression, reduced autophagic vacuoles, brain natriuretic peptide (BNP), high sensitivity troponin I, and left ventricular mass and wall thickness. In addition, there was improvement in symptoms, as assessed by New York Heart Association class and quality of life, as measured by the Kansas City Cardiomyopathy Questionnaire. Notably, the improvements and stabilization of BNP in Phase 1 patients were in direct contrast to worsening patterns observed in patients enrolled in a concurrent, prospective natural history study. The results demonstrated improvements and/or normalization across multiple quantifiable parameters that cardiologists use in clinical practice to enable risk assessment and treatment decisions.
About EMA PRIME Designation
Priority Medicines (PRIME) designation was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicines that target an unmet medical need and demonstrate the potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offers enhanced early interaction with companies developing promising medicines, to optimize development plans and speed up evaluation. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or that benefit patients without treatment options.