Talk Bio

The U.S. Food and Drug Administration (FDA) has expanded the approval of Vertex's Casgevy (exagamglogene autotemcel), a gene‑editing therapy, to include patients as young as 2 years of age with either sickle cell disease (SCD) with recurrent vaso‑occlusive crises (VOCs) or transfusion‑dependent β‑thalassemia (TDT). Casgevy was previously approved only for patients aged 12 years and older, so this marks a significant lowering of the minimum treatment age. The FDA based the expansion on clinical‑study data in children aged 5–under‑12 and extrapolated safety and efficacy to support use down to 2 years. This makes Casgevy the first gene therapy approved for children as young as 2 with SCD and also the first approved genetic therapy for very young children with both SCD and transfusion‑dependent β‑thalassemia. Vertex estimates that about 5,500 additional pediatric patients in the United States now become eligible for this one‑time treatment. The company is also pursuing regulatory revie...