FDA Approves First Gene Therapy Technology to Treat Children With Sickle Cell Disease

WASHINGTON — The Food and Drug Administration Wednesday granted supplemental approval for a breakthrough CRISPR gene therapy medication, making it the first available to treat young children with sickle cell disease.

Casgevy (exagamglogene autotemcel) was first approved in December 2023 as the first medication capable of treating sickle cell disease and transfusion-dependent beta-thalassemia, another hemoglobin-related condition. 

It was originally approved for patients aged 12 or older. With Wednesday’s supplemental approval, it is now available to children as young as 2 years old.