Benzinga | Mar 29, 2021 07:11AM EDT

Rocket Pharmaceuticals Receives EMA Priority Medicines Designation for RP-L201 Gene Therapy for Treatment of Leukocyte Adhesion Deficiency-I

Rocket 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.

"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."

The 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.

The ongoing, non-randomized, open-label Phase 1/2 study of RP-L201 recently completed enrollment. It is designed to evaluate the safety and efficacy of lentiviral vector (LVV)-based RP-L201 in pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. Data from the study presented at the 62nd American Society of Hematology Annual Meeting demonstrate evidence of safety and efficacy in three pediatric patients with severe LAD-I. These patients have shown sustained CD18 expression exceeding the 4-10% threshold associated with survival into adulthood and similarly encouraging peripheral blood vector copy numbers. RP-L201 was well tolerated with no drug product safety issues reported with infusion or post-treatment. The study is being conducted at the University of California Los Angeles, University College London (UCL)/Great Ormond Street Children's Hospital, and Hospital Infantil Universitario Ni?o Jes?s.

Further information about the RP-L201 clinical program is available here.