Rocket Pharmaceuticals, Inc, 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 U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the Company’s investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown.
“Receiving RMAT designation from the FDA for RP-L301 is a major achievement in our pursuit to bring the first, potentially curative gene therapy treatment to patients living with PKD who have high unmet need. Notably, PKD has an estimated prevalence of up to 8,000 patients in the U.S. and Europe and represents one of the most significant patient opportunities in our LV hematology portfolio,” said Kinnari Patel, PharmD, MBA, President and Chief Operating Officer, Rocket Pharma.
Dr. Patel continued, “Results from the RP-L301 program demonstrate robust efficacy in both adult patients for up to 30 months with a highly favorable safety profile and were recently presented at ASGCT. The first pediatric patient has shown promising initial results similar to the adults, and enrollment has been completed in the Phase 1 study. We look forward to initiating the Phase 2 pivotal trial in the fourth quarter of 2023 as we continue to advance our world-class pipeline for patients facing such rare and devastating diseases.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.
Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early FDA interactions to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.
SOURCE: Businesswire