Vertex Pharmaceuticals Announces Positive Long-Term Data for CASGEVY® at EHA Congress, Expanding Global Access for Sickle Cell Disease and Beta Thalassemia Patients
Reuters
Jun 12, 2025
Vertex Pharmaceuticals Announces Positive Long-Term Data for CASGEVY® at EHA Congress, Expanding Global Access for Sickle Cell Disease and Beta Thalassemia Patients
Vertex Pharmaceuticals Incorporated has announced the presentation of longer-term data for CASGEVY® (exagamglogene autotemcel) at the 2025 European Hematology Association (EHA) Congress. The data, derived from global clinical trials involving patients with severe sickle cell disease $(SCD)$ or transfusion-dependent beta thalassemia (TDT), underscore the durability of CASGEVY's clinical benefits. In SCD trials, 95.6% of evaluable patients were free from vaso-occlusive crises for at least 12 consecutive months. The longest follow-up extends over 5.5 years for SCD patients and over 6 years for TDT patients. CASGEVY is noted as the first approved CRISPR/Cas9 gene-edited therapy, with multiple reimbursement agreements in place to enhance global access.
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