FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

The FDA approved a supplemental indication for Casgevy (exagamglogene autotemcel), a CRISPR-based gene therapy, expanding its use to children aged 2 years and older with sickle cell disease or transfusion-dependent β thalassemia. The approval was granted in 53 days under the FDA's Commissioner's National Priority Voucher pilot program, based on clinical trial data showing efficacy in eliminating vaso-occlusive crises and achieving transfusion independence.