FDA expands use of first sickle cell disease gene therapy for children as young as 2 years

The U.S.

Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients ages 2 and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT).

Supplemental approval was granted to Vertex Pharmaceuticals. ...