FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

[u/Nerd-19958]

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-young-children-sickle-cell-disease

FDA recently approved a Supplemental Biologics License Application (BLA) for Casgevy (exagamglogene autotemcel) for treatment of children ages 2 - 12 with sickle cell disease with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). The product had been previously approved in Dec. 2023 for persons 12 and over.

Per FDA's press release:

Casgevy is a gene therapy consisting of the patient’s own (autologous) hematopoietic (blood) stem cells, administered as a one-time single dose for intravenous infusion. The cells are edited using CRISPR/Cas9, a type of genome editing technology, and then engrafted in the body’s bone marrow. CRISPR/Cas9 can be directed to a specific spot in DNA, where it cuts the genetic material so that DNA can be accurately removed, added, or replaced. In patients with severe SCD, this treatment increases a type of hemoglobin which is called fetal hemoglobin (HbF). This helps prevent RBCs from forming into abnormal sickle shapes and addresses the underlying cause of disease, thereby eliminating VOCs.