Business
Rocket Pharmaceuticals Receives EMA Priority Medicines (PRIME) Designation for RP-L201 Gene Therapy for Treatment of Leukocyte Adhesion Deficiency-I
— PRIME Designation to Facilitate Greater EMA Collaboration for Development and Potential Accelerated Regulatory Review in Europe — — LAD-I Program Now Holds
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n— PRIME Designation to Facilitate Greater EMA Collaboration for Development and Potential Accelerated Regulatory Review in Europe —\n\n— LAD-I Program Now Holds All Available Accelerated Regulatory Designations in the U.S. and EU —\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-L201, the Company’s investigational gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I). PRIME designation was granted based on encouraging preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trial of RP-L201.\n\n“We are delighted that the EMA has awarded PRIME designation to RP-L201 for the treatment of LAD-I. PRIME completes the full complement of U.S. and EU accelerated regulatory designations for RP-L201 and signals that regulators recognize the high unmet medical need in treating this devastating pediatric disease and our gene therapy’s potential to address the root cause of this disorder,” said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket. “More than half of LAD-I patients suffer with a severe variant in which mortality occurs in up to 75% of children prior to age two in the absence of a successful bone marrow transplant. Securing all possible accelerated designations will enable us to collaborate with both the FDA and EMA to speed the delivery of a potentially curative option for these patients. We look forward to sharing initial Phase 2 data from our potentially registration-enabling LAD-I trial in the second quarter of 2021.”\n\nThe PRIME program aims to optimize development plans and speed up evaluation of medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. These medicines are considered priority medicines by the EMA and are intended to reach patients earlier. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data.\n\nThe ongoing, non-randomized, open-label Phase 1/2 study of RP-L201 recently completed enrollment. It is designed to evaluate the safety and effic...