FDA expands use of first sickle cell disease gene therapy for children as young as 2 years

🇺🇸 Medical Xpress (US) —
FDA expands use of first sickle cell disease gene therapy for children as young as 2 years

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The U.S. Food and Drug Administration has expanded approval for Casgevy (exagamglogene autotemcel), a gene therapy for sickle cell disease and transfusion-dependent β thalassemia, now authorized for children as young as two years old. The supplemental approval was granted to Vertex Pharmaceuticals, expanding treatment options for young patients with these blood disorders.

The U.S. Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients ages 2 and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). Supplemental approval was granted to Vertex Pharmaceuticals.

Health FDA Casgevy gene therapy sickle cell disease β thalassemia Vertex Pharmaceuticals

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