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

The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exa-cel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD.