On July 1, 2026, Vertex Pharmaceuticals announced that the FDA approved an expanded indication for CASGEVY (exagamglogene autotemcel) to treat children aged two years and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises or who have transfusion-dependent beta thalassemia (TDT). The approval makes CASGEVY the first CRISPR-based gene-editing therapy approved for children aged two years and older with SCD or TDT.
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth.
CASGEVY was originally approved in the United States in 2023 for patients 12 years and older. The supplemental application was reviewed under the Commissioner’s National Priority Voucher—a pilot program launched by the FDA in June 2025 to accelerate review of drug and biological product applications that align with national health priorities.
“Earlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold,” said Haydar Frangoul, M.D., M.S., Member of Vertex’s SCD Program Steering Committee.
The expanded approval comes less than one month after Vertex presented European data in children aged five to eleven years with SCD and TDT. According to Vertex, the results demonstrated efficacy and safety consistent with those previously observed in adult and adolescent patients. We previously reported on Vertex’s presentation of similar data in the United States.
Vertex also recently submitted regulatory applications in the United Kingdom and the Kingdom of Saudi Arabia seeking to expand CASGEVY’s indication to children aged five years and older.
CASGEVY is currently available at more than 75 independently operated authorized treatment centers throughout the United States.