CASGEVY is approved by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs). The use of CASGEVY for the treatment of TDT in the U.S. remains investigational. Vertex has submitted a BLA to the U.S. FDA for the potential use of CASGEVY for patients 12 years and older with TDT and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 30, 2024.