FDA reviewing Casgevy for children with sickle cell as young as 5

The U.S. Food and Drug Administration is reviewing an application from Vertex Pharmaceuticals seeking approval of Casgevy (exagamglogene autotemcel) for children with sickle cell disease (SCD) as young as 5 years old.

The gene-editing therapy is currently approved in the U.S. and elsewhere for people ages 12 and older with SCD and recurrent vaso-occlusive crises (VOCs).

Vertex’s new application was submitted using a Commissioner’s National Priority Voucher, which shortens a standard 10-month review time down to one or two months. Similar applications have been submitted in the U.K. and Saudi Arabia.