Vertex Presents New Data on CASGEVY, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory Submissions

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data demonstrating the clinical benefits of CASGEVY (exagamglogene autotemcel) in people ages 5 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 the transformative profile established in adult and adolescent patients. These 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 Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

“Despite optimized supportive therapy, children living with sickle cell disease and transfusion‑dependent beta thalassemia carry a significant disease burden from a very young age, with progressive complications leading to the irreversible and life-shortening consequences of these diseases,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair of Vertex’s TDT Program Steering Committee, and presenting author of the 5–11 years old CASGEVY data at EHA. “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 clinical data for children ages 5–11 presented at EHA and published in NEJM

Data from an interim analysis of the CLIMB-151 and CLIMB-141 studies highlight the transformative potential CASGEVY can provide to children ages 5–11 and are consistent with the durable clinical profile established in adult and adolescent patients. Collectively, these findings highlight the potential benefits of addressing vaso-occlusive crises (VOCs) and transfusion burden earlier in life, which can begin in childhood and are associated with cumulative, long-term complications in SCD and TDT including organ damage.

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