Reported Saturday, Vertex Announces Pivotal CASGEVY Studies In Children Ages 5-11 Years Showing Transformative Potential And Consistent Efficacy And Safety Versus Ages 12 Years And Older

Benzinga · 2d ago
  • - Data from pivotal studies of CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrates the transformative potential of the therapy in younger patients -
  • - Efficacy and safety data in children 5-11 years are consistent with the durable and positive benefit/risk profile established from clinical studies in patients 12 years of age and older -
  • - Vertex expects to initiate global regulatory submissions for CASGEVY in children 5-11 years in 1H 2026 -