Rocket’s LAD-I Gene Therapy BLA Accepted for Priority Review


The therapy has a PDUFA date of March 31, 2024.

The FDA has accepted and granted Priority Review to Rocket Pharmaceuticals’ biologics license application (BLA) for the gene therapy RP-L201 (marnetegragene autotemcel) intended for treated of severe leukocyte adhesion deficiency-I (LAD-I) with a Prescription Drug User Fee Act (PDUFA) target action date of March 31, 2024.1

“Today’s acceptance of the BLA by the FDA marks a significant milestone for Rocket towards our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I. For these patients, survival beyond childhood is uncommon. Bone marrow transplant is currently the only treatment option, has substantial morbidity and mortality, and may not be available in time for these children,” Kinnari Patel, PharmD, MBA, president and chief operating officer, Rocket Pharma, said in a statement.1 “We are incredibly grateful to the patients, caregivers and researchers who have shared this journey with us and look forward to continuing our close collaboration with the FDA during the review period as we work to bring RP-L201 to patients as quickly as possible.”

RP-L201 is an autologous, gene-edited stem cell therapy that delivers a functional copy of the ITGB2 gene, mutations in which cause LAD-I, through the use of a lentiviral vector. The ITGB2 gene encodes for beta-2 integrin component CD18 protein that facilitates leukocyte adhesion and helps fight infections. Infants and children with severe LAD-I experience recurrent, life-threatening infections and often do not survive past childhood without allogeneic hematopoietic stem cell transplant.

READ MORE: Rocket Pharmaceuticals Confirms Phase 2 Trial Plans for Danon Disease Gene Therapy With FDA

RP-L201 has received Regenerative Medicine Advanced Therapy, Rare Pediatric, Orphan Drug, and Fast Track designations in the United States (US); and PRIME, Advanced Therapy Medicinal Product, and Orphan Drug designations in the European Union. Rocket announced that it had submitted the BLA for RP-L201 in August 2023.2 The program is funded by a grant from the California Institute for Regenerative Medicine.

“As the Principal Investigator in the US, I oversaw treatment of 6 of the 9 LAD-I patients in this trial. In my opinion, the results are remarkable. All of these children have been in good health with no significant LAD-I-related infections or inflammatory skin lesions since treatment. Based on what I see, they are all experiencing a normal childhood life, which is the goal of this type of potentially curative gene therapy,” Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology, University of California, Los Angeles (UCLA) and director, UCLA Human Gene and Cell Therapy Program, added.1

The latest data on RP-L201 from its phase 2 trial (NCT03812263) were presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in May 2023 and showcased follow-up data of 12 to 24 months.3 All 9 patients had a 100% overall survival rate with evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety profile continues to be favorable with no treatment-related serious adverse events (AEs) and AEs consistent with study procedures including busulfan conditioning.

1. Rocket Pharmaceuticals announces FDA acceptance of biologics license application with priority review for RP-L201 (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). News release. Rocket Pharmaceuticals.
2. Rocket Pharmaceuticals reports second quarter 2023 financial results and highlights recent progress. News release. Rocket Pharmaceuticals. August 10, 2023.
3. Rocket Pharmaceuticals presents positive data from LV hematology and AAV cardiovascular gene therapy programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). News release. Rocket Pharmaceuticals. May 19, 2023.
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