FDA Approves Casgevy Gene Therapy for Children as Young as 2 Years Old

The U.S. Food and Drug Administration (FDA) has approved an expanded indication for Vertex Pharmaceuticals' gene therapy Casgevy, allowing its use in children as young as 2 years old with inherited blood disorders, including sickle cell disease.

Casgevy is a one-time gene therapy created using the patient's own hematopoietic stem cells. It was previously approved for patients aged 12 years and older with sickle cell disease and transfusion-dependent beta-thalassemia.

In clinical trials involving children aged 5 to 12 years with sickle cell disease, all eight patients remained free of severe vaso-occlusive pain crises for at least 12 months following treatment.

Among children with transfusion-dependent beta-thalassemia, eight of nine patients achieved transfusion independence for one year or longer, with a median transfusion-free duration of 20.1 months.

The FDA granted the expanded approval under its Commissioner's National Priority Voucher program, which significantly accelerates the review of selected therapies. The decision was made just 53 days after the application was submitted.

The FDA first approved gene therapies developed by Vertex Pharmaceuticals and CRISPR Therapeutics for adolescents and adults aged 12 years and older with sickle cell disease in 2023.

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