FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

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Summary

FDA approves Casgevy (exagamglogene autotemcel) as a gene therapy for young children aged 2 years and older with sickle cell disease and recurrent VOCs, or tran…

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Diseases / indications

• Sickle Cell Disease
• Transfusion-dependent Thalassemia

Drugs mentioned

• Casgevy (exagamglogene autotemcel)

Snippet

The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT).