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

Why it matters locally: This expanded FDA approval for Casgevy will directly impact Massachusetts families with young children suffering from sickle cell disease, offering a new treatment option for a debilitating genetic disorder.

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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).