Vertex Pharmaceuticals presents new data on CASGEVY at ASH

Vertex Pharmaceuticals (VRTX) announced data from multiple studies demonstrating the clinical benefits of CASGEVY in people ages 5 years and older living with severe sickle cell disease, SCD, or transfusion-dependent beta thalassemia, TDT. The results, including the first presentation of clinical data from pivotal studies in children ages 5-11 years, and longer-term data from the pivotal studies of people with severe SCD and TDT ages 12 years and older, will be presented at the American Society of Hematology, ASH, Annual Meeting. Key points: 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