Gene therapy for children with sickle cell disease approved by FDA

Gene therapy for children with sickle cell disease approved by FDA

A new treatment for children aged 2 or older with sickle cell disease has been approved by the U.S. Food & Drug Administration.

FDA expands sickle cell gene therapy to patients as young as 2

Casgevy (exagamglogene autotemcel) is now approved in the U.S. to treat children as young as 2 with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) initially approved the Vertex Pharmaceuticals’ gene-editing therapy in 2023 for SCD patients ages 12 and older who experienced recurrent painful episodes known as VOCs. With this decision, Casgevy becomes the first gene-editing therapy…

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

The US FDA issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged two years and older with either sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia. [US FDA] Press Release