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Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)
CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":" CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket 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 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. RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 RP-L301 clinical trial and its potential to cure a life-threatening disease for which no curative therapies currently exist. The designation will provide the benefits of added FDA guidance and expedited review through the program’s development.\n\n\n“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. “Further, all four Rocket-sponsored programs with clinical data now have received RMAT designation from the FDA across both platforms, a unique showcase of our team’s ability both to select appropriate targets and develop gene therapies for them.”\n\n\nDr. 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.”\n\n\nEstablished under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline ...