FDA Expands Casgevy Approval to Children as Young as 2 Years Old

The FDA approved a supplemental Biologics License Application expanding Casgevy (exagamglogene autotemcel) to patients 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia — down from the prior floor of 12 years old. It is the first gene therapy approved for children this young with sickle cell disease, opening eligibility to an estimated 5,500 additional U.S. children. Efficacy and safety in patients 5 to under 12 with TDT were evaluated in a 15-patient trial; 8 of 9 efficacy-evaluable TDT patients achieved transfusion independence for 12 consecutive months (median duration 20.1 months). Vertex Pharmaceuticals and CRISPR Therapeutics jointly develop and market Casgevy, the world's first approved CRISPR-based medicine.