FDA Expands Use of First Sickle Cell Disease Gene Therapy for Children as Young as 2 Years

WEDNESDAY, July 8, 2026 (HealthDay News) -- The U.S. Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). 

Casgevy was previously approved for the treatment of patients 12 years and older with SCD with recurrent VOCs or TDT and is now the first gene therapy approved for patients aged 2 years and older. Therapy consists of autologous hematopoietic stem cells, administered as a one-time single dose via intravenous infusion. Cells are edited using CRISPR/Cas9 genome editing technology and then engrafted in the body's bone marrow.