• Drug Coverage
  • Hypertrophic Cardiomyopathy (HCM)
  • Vaccines: 2023 Year in Review
  • Eyecare
  • Urothelial Carcinoma
  • Women's Health
  • Hemophilia
  • Heart Failure
  • Vaccines
  • Neonatal Care
  • Type II Inflammation
  • Substance Use Disorder
  • Gene Therapy
  • Lung Cancer
  • Spinal Muscular Atrophy
  • HIV
  • Post-Acute Care
  • Liver Disease
  • Pulmonary Arterial Hypertension
  • Safety & Recalls
  • Biologics
  • Asthma
  • Atrial Fibrillation
  • Type I Diabetes
  • RSV
  • COVID-19
  • Cardiovascular Diseases
  • Breast Cancer
  • Prescription Digital Therapeutics
  • Reproductive Health
  • The Improving Patient Access Podcast
  • Blood Cancer
  • Ulcerative Colitis
  • Respiratory Conditions
  • Multiple Sclerosis
  • Digital Health
  • Population Health
  • Sleep Disorders
  • Biosimilars
  • Plaque Psoriasis
  • Leukemia and Lymphoma
  • Oncology
  • Pediatrics
  • Urology
  • Obstetrics-Gynecology & Women's Health
  • Opioids
  • Solid Tumors
  • Autoimmune Diseases
  • Dermatology
  • Diabetes
  • Mental Health

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.,

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.

Related Videos
Related Content
© 2024 MJH Life Sciences

All rights reserved.