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

Why it matters locally: While the prevalence of sickle cell disease in Alaska is lower than in some other states, this FDA approval provides a new treatment option for the small number of Alaskan children affected by this severe 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).