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
- Data from pivotal studies of CASGEVY in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrate transformative potential in younger patients, consistent with the durable benefits established in patients 12 years and older -
- Data simultaneously published in the New England Journal of Medicine -
- Regulatory review underway in the United States to expand the use of CASGEVY, and Vertex has recently completed submissions in the Kingdom of Saudi Arabia and United Kingdom -
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).
