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.
Listen
Navitus to Offer Unbranded Stelara Biosimilar, Remove Stelara from Formulary
March 13th 2025Lumicera Health Services, Navitus’ specialty pharmacy, has made a deal with Teva to offer an unbranded biosimilar that they estimate will save $112,000 and $336,000 per patient per year. Navitus will remove Stelara from formulary on July 1, 2025.
Read More