Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the U.S. Food and Drug Administration (FDA) has approved expanded use of CASGEVY^®(exagamglogene autotemcel) for the treatment of people ages 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first approved genetic therapy indicated for children as young as 2 years for both SCD and TDT.

“Just as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia. The remarkable consistency of results across age groups reinforces the potential of CASGEVY to deliver durable, transformative benefits to those who have historically had limited options,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We’re deeply grateful to the patients, families and investigators who participated in the clinical trials that led to this historic approval, and we are ready to bring CASGEVY to children and their families across the U.S.”

“Today’s approval offers renewed hope for children living with sickle cell disease or transfusion‑dependent beta thalassemia,” said Haydar Frangoul, M.D., M.S., Medical Director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, investigator with Sarah Cannon Research Institute (SCRI) and Member of Vertex’s SCD Program Steering Committee. “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.”

Vertex has established a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to eligible patients through existing access and reimbursement pathways