FDA expands Casgevy approval to children as young as two

2 July 2026

The US Food and Drug Administration (FDA) has expanded approval of Vertex Pharmaceuticals’ (Nasdaq: VRTX) Casgevy (exagamglogene autotemcel) to patients aged two years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).

This decision lowers the previous age threshold from 12 years and older, making Casgevy the first gene therapy approved by the FDA for patients as young as two with SCD.

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