India, July 2 -- Vertex Pharmaceuticals Inc. (VRTX) announced that the U.S. FDA has approved expanded use of CASGEVY for patients ages 2 years and older with sickle cell disease experiencing vaso-occlusive crises or transfusion-dependent beta thalassemia.
CASGEVY is now the first genetic therapy authorized for children as young as 2 years for both conditions, broadening access beyond the prior approval for patients 12 and older. This expansion makes approximately 5,500 additional children in the U.S eligible for the one-time treatment.
CASGEVY is a gene-edited autologous stem cell therapy designed to modify a patient's own hematopoietic stem cells to produce high levels of fetal hemoglobin (HbF). By increasing HbF, the therapy reduces the...