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

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• 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.

  60% confidence

• Efficacy and safety outcomes of CASGEVY in children ages 5–11 are consistent with the transformative profile established in adult and adolescent patients.

  60% confidence

• 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 irreversible and life-shortening consequences, even with optimized supportive therapy.

  60% confidence

• The CASGEVY pediatric data represent a profoundly important step forward, with the possibility of providing earlier intervention to prevent complications in children, for families who have had limited potentially curative options.

  60% confidence

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