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

Why it matters locally: South Carolina has a higher prevalence of sickle cell disease compared to the national average, making this FDA approval particularly relevant for families and healthcare providers across the state who treat young children with the condition.

The U.S. Food and Drug Administration (FDA) today announced a supplemental approval for Casgevy (exagamglogene autotemcel). This expanded approval permits the use of the gene therapy for patients aged two years and older. The therapy is indicated for individuals diagnosed with sickle cell disease (SCD) who experience recurrent vaso-occlusive crises (VOCs). Additionally, the approval covers patients with transfusion-dependent beta thalassemia (TDT).