FDA Assigns Review Date for Gene Therapy for Rare Immune Disorder
FDA has set an action date of March 31, 2024, for marnetegragene autotemcel to treat infants with a serious and often fatal immunodeficiency.
The FDA has granted priority review for Rocket Pharmaceuticals’ biologics license application (BLA) for marnetegragene autotemcel (RP-L201) to treat the rare, autosomal recessive pediatric disease leukocyte adhesion deficiency-I (LAD-I). Regulators set a Prescription Drug User Fee Act (PDUFA) date set of March 31, 2024.
Severe leukocyte adhesion deficiency-I is a caused by mutations in the ITGB2 gene that encodes for CD18, a key protein that facilitates the immune response against infections. As a result, white blood cells (leukocytes) do not function normally. Children with this disease experience life-threatening bacterial and fungal infections that respond poorly to antibiotics. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. LAD-I is estimated to impact an estimated 800 to 1,000 children in the United States and Europe.
Marnetegragene autotemcel is a lentiviral vector-based investigational gene therapy. It contains autologous hematopoietic stem cells that have been genetically modified to deliver a functional copy of the ITGB2 gene.
Data from the global phase 1/2 study of marnetegragene autotemcel demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 12 to 24 months of available follow-up.
Donald B. Kohn, M.D.,
“As the principal investigator in the United States, I oversaw treatment of six of the nine 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,” Donald B. Kohn, M.D., distinguished professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and director of the UCLA Human Gene and Cell Therapy Program, said in a press release.
Data also showed large decreases compared with pretreatment history in the incidences of significant infections, combined with evidence of resolution of LAD-I-related skin lesions and restoration of wound repair capabilities. It was very well tolerated in all patients with no treatment-related adverse events.
In this episode of the "Meet the Board" podcast series, Briana Contreras, Managed Healthcare Executive editor, speaks with Ateev Mehrotra, a member of the MHE editorial advisory board and a professor of healthcare policy and medicine at Harvard Medical School. Mehtrotra is also a hospitalist at the Beth Israel Deaconess Medical Center in Boston. In the discussion, Contreras gets to know Mehrotra more on a personal level and picks his brain on some of his research interests including telehealth, alternative payment models and price transparency.
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