Vertex Pharmaceuticals Incorporated has announced new clinical data on CASGEVY® (exagamglogene autotemcel) in children aged 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia. The data, which were presented at the American Society of Hematology $(ASH)$ Annual Meeting, are the first-ever clinical results for any genetic therapy in this age group. The efficacy and safety outcomes in children aged 5-11 years were consistent with previously established data in patients aged 12 years and older. Vertex reported that enrollment and dosing for the 5-11 years cohort are complete, and the company plans to initiate global regulatory submissions, including a supplemental Biologics License Application in the U.S., in the first half of 2026. CASGEVY is currently approved for eligible patients aged 12 years and older in multiple countries.
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