Vertex Pharmaceuticals has announced new clinical data highlighting the benefits of CASGEVY (exagamglogene autotemcel) in children aged 5–11 living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
The findings, presented at the European Hematology Association Congress and published in the New England Journal of Medicine, showed strong efficacy and a safety profile consistent with results previously seen in adolescents and adults.
In the Phase 3 CLIMB-151 study, all children with sufficient follow-up remained free from vaso-occlusive crises for at least 12 consecutive months. In the Phase 3 CLIMB-141 study, all evaluable children with TDT achieved transfusion independence for at least 12 months.
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Vertex also announced ongoing efforts to expand access to CASGEVY. Regulatory review is underway in the United States for younger children, while submissions have recently been completed in Saudi Arabia and the United Kingdom.
CASGEVY is currently approved in multiple countries for eligible patients aged 12 years and older with sickle cell disease or transfusion-dependent beta thalassemia. The use of CASGEVY in children aged 5–11 remains investigational.
The company said the latest results support the potential of earlier intervention to reduce disease burden and improve long-term outcomes for patients living with these inherited blood disorders.
