Vertex Secures US FDA Approval for Expanded Use of CASGEVY in Young Children
Thursday, July 02, 2026
Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) to expand the use of CASGEVY® (exagamglogene autotemcel) for patients aged two years and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassaemia (TDT).
The approval makes CASGEVY the first authorised genetic therapy for children as young as two years with both sickle cell disease and transfusion-dependent beta thalassaemia in the United States.
The expanded indication is supported by consistent clinical results across different age groups, reinforcing the therapy's potential to provide long-term benefits for patients with these inherited blood disorders.
Vertex has established a network of independently operated authorised treatment centres (ATCs) across the United States to provide CASGEVY to eligible patients through existing access and reimbursement pathways. More than 75 treatment centres are currently active and offering the therapy.
Source: businesswire.com
