The US Food and Drug Administration (FDA) has expanded approval of exagamglogene autotemcel (exa-cel; Casgevy) to children ≥ 2 years of age with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT), making it the first CRISPR-based gene-editing therapy approved for children this young with either condition.
Announced on July 1, 2026, the supplemental approval lowers the minimum approved age from 12 years and expands access to a one-time gene-editing therapy earlier in the disease course. The decision was supported by efficacy and safety data in children aged 5 to younger than 12 years, with the indication extending to children aged 2 to younger than 5 years based on extrapolation from those findings. The approval was granted 53 days after filing through the FDA Commissioner's National Priority Voucher pilot program.