Vertex Pharmaceuticals Announces Positive Long-Term Data for CASGEVY® at EHA Congress, Expanding Global Access for Sickle Cell Disease and Beta Thalassemia Patients

Reuters
2025/06/12
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.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Vertex Pharmaceuticals Incorporated published the original content used to generate this news brief on June 12, 2025, and is solely responsible for the information contained therein.

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