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

Core Insights - Vertex Pharmaceuticals announced clinical data demonstrating the benefits of CASGEVY (exagamglogene autotemcel) for patients aged 5 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT) [1][2] - The data includes the first clinical results for children aged 5-11 years and long-term outcomes for patients aged 12 years and older, to be presented at the American Society of Hematology (ASH) Annual Meeting [1][4] Company Overview - Vertex is a global biotechnology company focused on creating transformative medicines for serious diseases, including SCD and TDT [28][29] - The company has a robust pipeline of investigational therapies across various serious diseases, leveraging deep insights into human biology [28] Product Information - CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy that aims to increase fetal hemoglobin (HbF) levels in patients with SCD and TDT [8][14] - The therapy has shown potential to reduce or eliminate vaso-occlusive crises (VOCs) in SCD patients and the need for regular blood transfusions in TDT patients [8][14] Clinical Study Results - In the Phase 3 CLIMB-151 study for children with SCD, all patients with sufficient follow-up achieved the primary endpoint of being free from VOCs for at least 12 consecutive months [5] - In the Phase 3 CLIMB-141 study for children with TDT, all patients with sufficient follow-up achieved transfusion independence for at least 12 consecutive months while maintaining a hemoglobin level of at least 9 g/dL [5] - Long-term data for patients aged 12 years and older showed that 100% of SCD patients achieved VOC-free status for a mean duration of 35.3 months, while 98.2% of TDT patients achieved transfusion independence for a mean duration of 41.4 months [4] Regulatory and Future Plans - Vertex plans to initiate global regulatory filings for CASGEVY in the 5-11 age group in the first half of next year, having received a Commissioner's National Priority Voucher from the FDA to expedite the review process [12] - The company aims to extend studies to include younger children aged 2-4 years [10][12]