FDA Grants First Gene-Therapy Approval for Children 2+ with Sickle Cell Disease

The U.S. Food and Drug Administration issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged two years and older who have sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia.

This is the first gene-therapy approval for children as young as two with sickle cell disease, expanding the earlier approval that was limited to patients twelve years and older.

The agency highlighted its use of expedited review programs, including the National Priority Voucher pilot, to accelerate access to treatments for diseases with significant unmet medical needs while maintaining rigorous safety standards.

The decision adds a new therapeutic option for a condition that causes severe health complications and has historically had limited treatment choices.