Press release
Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions
- Data from pivotal studies of CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrates the
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- 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 -\n\n- 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 -\n\n- Vertex expects to initiate global regulatory submissions for CASGEVY in children 5-11 years in 1H 2026 -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies 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, 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. CASGEVY is currently approved for eligible people ages 12 years and older with SCD or TDT in the United States, Great Britain, the European Union, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Kuwait, Qatar, Canada, Switzerland and the United Arab Emirates.\n\n“These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the transformative potential of CASGEVY,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With dosing completed in the 5-11 age group and the Commissioner’s National Priority Voucher for CASGEVY in this population in hand, we are excited to begin global regulatory filings in the first half of next year and bring this potentially transformative therapy to eligible children as soon as possible.”\n\n“As an investigator in the clinical program for patients 12 years and older and after having real-world experience with CASGEVY as an early commercial treatment center, I have seen firsthand the transformative impact this therapy has had on older patients with SCD or TDT. I am excited to hopefully be able to offer this optio...