— Casgevy also now indicated for transfusion-dependent beta thalassemia in children 2 years and up
July 2, 2026
• 2 min read
The FDA on Wednesday expanded the approval for exagamglogene autotemcel (exa-cel, Casgevy) in severe sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta thalassemia to include children 2 years and up.
Exa-cel, the first FDA-approved therapy utilizing CRISPR/Cas9 gene editing technology, was previously only approved for adults and pediatric patients 12 years and older. The expanded indication makes exa-cel the first gene therapy approved for kids as young as 2 with these debilitating diseases.
