FDA widens Vertex’s Casgevy use for paediatric patients - Pharmaceutical Technology

Children as young as two with either sickle cell disease or thalassemia are now eligible for the gene therapy. Children as young as two with either sickle cell disease or thalassemia are now eligible for the gene therapy. Casgevy generated global revenue of $116m in 2025, representing 64 patients receiving infusions. Credit: JHVEPhoto/Shutterstock.com. The US Food and Drug Administration (FDA) has approved expanded use of Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) in children with one of two types of inherited blood disorders, marking the first such treatment authorised in this age group. Vertex’s gene therapy was already approved in patients aged…

Source: Pharmaceutical Technology