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Reported Earlier, Vertex Wins FDA Approval Expanding CASGEVY Use To Children As Young As 2 With Severe Sickle Cell Disease Or Beta Thalassemia

Benzinga · 2d ago

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- First and only approved genetic therapy to treat children as young as 2 years for both severe sickle cell disease and transfusion-dependent beta thalassemia -

- Approximately 5,500 additional children in the U.S. are now eligible for this established one-time therapy, expanding upon the prior FDA approval in people 12 years and older -

- Regulatory review for label expansion underway in the Kingdom of Saudi Arabia and United Kingdom

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