Vertex Pharmaceuticals presents long-term data on CASGEVY

Vertex Pharmaceuticals (VRTX) recently announced longer-term data for PrCASGEVY from global ongoing pivotal clinical trials in people with severe sickle cell disease or transfusion-dependent beta thalassemia. The results, presented at the European Hematology Association Congress, continue to demonstrate the durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first authorized CRISPR/Cas9 gene-edited therapy. In SCD, 43/45 evaluable patients were free from vaso-occlusive crises for at least 12 consecutive months in CLIMB-121 and CLIMB-131 combined. The mean duration of VOC-free was 35.0 months. All evaluable patients achieved freedom from in-patient hospitalization for severe VOCs for at least 12 consecutive months in CLIMB-121 and CLIMB-131 combined with a mean hospitalization-free of 36.1 months. In TDT, 54/55 evaluable patients achieved transfusion-independence for at least 12 consecutive months with a weighted average hemoglobin of at least 9 g/dL in CLIMB-111 and CLIMB-131 combined. The mean duration of transfusion independence was 40.5 months. The one evaluable patient who did not achieve TI12 has been transfusion free for 14.8 months. Iron removal therapy has been stopped for more than 6 months in 39/56 treated patients following infusion with CASGEVY, with sustained improvement in ferritin and liver iron content, suggesting that CASGEVY has the potential to correct ineffective erythropoiesis. Patients continue to demonstrate stable levels of fetal hemoglobin and allelic editing. The safety profile of CASGEVY continues to be generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, Denmark, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. In Canada, CASGEVY received positive recommendations for reimbursement from both Canadian health technology agencies between December 2024 and January 2025; however, a Letter of Engagement from the pan-Canadian Pharmaceutical Alliance (pCPA) is pending. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients.