FDA extends Casgevy to children aged 2 and up with sickle cell disease or beta thalassemia

CRISPR Therapeutics/Casgevy (exa-cel)

The FDA widened Casgevy's approval from age 12 down to age 2, the first gene therapy for sickle cell disease in children this young. In trials of children aged 5 to 11, all 8 sickle cell patients assessed went a year without severe pain crises and 8 of 9 beta thalassemia patients stopped needing transfusions. The review took 53 days under a priority voucher.