Vertex Says Pediatric CASGEVY Data Show Consistent Benefits In Rare Blood Disorders

Vertex Pharmaceuticals reported positive Phase 3 clinical data for CASGEVY in children ages 5-11 with severe sickle cell disease and transfusion-dependent beta thalassemia. All evaluated patients achieved primary endpoints with sustained clinical benefits and a consistent safety profile. The company is pursuing FDA approval to expand the therapy's use to younger children.