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

Why it matters locally: The expanded FDA approval for this gene therapy could significantly impact the estimated 1 in 16,300 Texans born with sickle cell disease, offering new treatment options for young patients and their families across the state.

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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).