On December 6, 2025, Vertex Pharmaceuticals Inc. (“Vertex”) announced data from several studies demonstrating the clinical benefits of CASGEVY® (exagamglogene autotemcel), including, for the first time, clinical data from studies in children ages 5-11 years. CASGEVY® is a non-viral, ex-vivo CRISPR/Cas9 gene-edited cell therapy that edits a patient’s own hematopoietic stem and progenitor cells at a specific region of the BCL11A gene. CASGEVY® is currently indicated as a one-time therapy in patients 12 years and older to treat patients with sickle cell disease that have frequent vaso-occlusive crises or for patients with β-thalassemia that need regular blood transfusions.
Vertex stated that it expects to initiate global regulatory filings for the 5–11 year age group in the first half of 2026, including the filing of a supplemental Biologics License Application (“sBLA”) in the United States. Vertex has received a Commissioner’s National Priority Voucher from the FDA for accelerated review of the sBLA once submitted.