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

Why it matters locally: While Casgevy is now approved for pediatric use, access to this specialized gene therapy in Nevada may depend on the availability of treatment centers and healthcare providers equipped to administer it.

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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).