FDA expands Casgevy approval to age 2

The U.S. United States — Food and Drug Administration issued a supplemental approval for Casgevy (exagamglogene autotemcel), expanding its use to patients aged 2 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD. Casgevy, developed by Vertex Pharmaceuticals, is a one-time gene therapy using CRISPR/Cas9 editing of the patient's own hematopoietic stem cells. The approval was based on clinical trials in patients aged 5 to less than 12 years, with extrapolation to younger children. The FDA completed its review in 53 days under the Commissioner's National Priority Voucher pilot program. The therapy also holds Orphan Drug, RMAT, and Fast Track designations.