Vertex Pharmaceuticals has announced data demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in people ages five years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).  

The results, from pivotal studies in children ages 5–11, show that the efficacy and safety outcomes in this age group are consistent with those established in adult and adolescent patients.  

The data were presented at the European Hematology Association (EHA) Congress and simultaneously published in the New England Journal of Medicine (NEJM). 

“The data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits Casgevy can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life,” said Dr Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. 

Franco Locatelli, Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, and Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, added: “These data represent a profoundly important step forward, and I look forward to the possibility of providing earlier intervention to prevent complications in children and for families who have had limited potentially curative options to date.” 

Casgevy is currently approved for eligible people 12 years and older with SCD with recurrent VOCs or TDT in several countries around the world. In the US the regulatory review is underway with the FDA to expand the use of Casgevy to younger children after Vertex was awarded the Commissioner’s National Priority Voucher.  

Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and UK to expand the use of Casgevy to younger children.

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