Vertex Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has expanded the approval of CASGEVY (exagamglogene autotemcel), allowing its use in children 2 years of age and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) and transfusion-dependent beta thalassemia (TDT). The therapy was previously approved only for patients aged 12 years and older.
The expanded indication makes CASGEVY the first and only approved genetic therapy available for children as young as two years with either of these severe inherited blood disorders in the United States. The decision enables eligible patients to receive treatment much earlier, potentially before irreversible organ damage develops.
CASGEVY is a one-time CRISPR/Cas9 gene-editing therapy developed by Vertex in collaboration with CRISPR Therapeutics. The treatment works by editing a patient’s own blood-forming stem cells to increase production of fetal hemoglobin (HbF), helping prevent sickling of red blood cells in sickle cell disease and reducing the need for chronic blood transfusions in beta thalassemia.
According to Vertex, the FDA’s decision was supported by clinical evidence demonstrating that the therapy’s safety profile and treatment benefits in younger children are consistent with those observed in older patients. The company believes earlier intervention could improve long-term health outcomes by reducing disease-related complications before they become permanent.
Vertex also stated that regulatory applications seeking similar pediatric label expansions are currently under review in several other countries, aiming to broaden global access to the gene-editing therapy.
