FDA expands use of first sickle cell disease gene therapy for children as young as 2 years

The U.S. Food and Drug Administration has issued a supplemental support for Casgevy (exagamglogene autotemcel) for patients ages 2 and older with either sickle compartment illness (SCD) with recurrent vaso-occlusive crises (VOCs) oregon transfusion-dependent β thalassemia (TDT). Supplemental support was granted to Vertex Pharmaceuticals.