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Vertex Says Long-Term Data Show Casgevy's 'Durable' Clinical Benefits in Blood Disorder Trials
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Vertex Says Long-Term Data Show Casgevy's 'Durable' Clinical Benefits in Blood Disorder Trials
Jul 3, 2025 11:17 AM

01:46 PM EDT, 07/03/2025 (MT Newswires) -- Vertex Pharmaceuticals ( VRTX ) said Thursday that longer term data from ongoing clinical trials show Casgevy's "durable" clinical benefits in people with severe sickle cell disease or transfusion-dependent beta thalassemia.

The company said the longest follow up now extends more than 5.5 years in sickle cell patients and over 6 years in thalassemia patients.

Among sickle cell patients 95.6% were free from vaso-occlusive crises for at least 12 straight months, and all avoided hospitalization during that period. These improvements lasted an average of about three years, the company said.

Among patients with transfusion-dependent beta thalassemia, Vertex said 98.2% achieved transfusion independence for at least 12 straight months, with stable hemoglobin levels. On average, they stayed transfusion-free for about 40 months, the company added.

The company also said it has reached reimbursement deals to make Casgevy available in several countries, including the US, the UK, and parts of Europe and the Middle East. In Canada, the drug has received positive funding recommendations, but final pricing agreements are still pending.

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