Casgevy approved for children as young as 2 years of age

The FDA issued a supplemental approval for exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals) for patients age 2 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT).

The decision makes exagamglogene autotemcel the first gene therapy approved for patients aged 2 years and older with SCD. Exagamglogene autotemcelwas previously approved for patients aged 12 years and older with SCD with recurrent VOCs or TDT. The approval was granted 53 days after filing and was the eighth approval selected for the FDA Commissioner’s National Priority Voucher pilot program.

Exagamglogene autotemcel consists of a patient’s own autologous hematopoietic stem cells and is administered as a one-time intravenous infusion. The cells are edited using CRISPR/Cas9 technology and then engrafted in the bone marrow. According to the FDA, full myeloablative conditioning is administered before treatment.

In patients with severe SCD, the treatment increases fetal hemoglobin, which helps prevent red blood cells from forming abnormal sickle shapes and addresses the underlying cause of disease, thereby eliminating VOCs. In patients with TDT, the treatment increases fetal hemoglobin and total hemoglobin levels, eliminating dependence on regular red blood cell transfusions.

The safety and effectiveness of exagamglogene autotemcel in patients aged 5 years to younger than 12 years with SCD were evaluated in a clinical trial that included 11 patients. All 8 patients evaluable for efficacy achieved the primary efficacy outcome of no protocol-defined severe VOCs for at least 12 consecutive months within the first 24 months after infusion. In a TDT trial of 15 patients aged 5 years to younger than 12 years, 8 of 9 efficacy-evaluable patients achieved transfusion independence for 12 consecutive months, with a median duration of 20.1 months. The FDA said extrapolation to patients aged 2 years and older was granted based on product characteristics and clinical study data.

Source(s): U.S. Food and Drug Administration. FDA approves first gene therapy for young children with sickle cell disease. News release. U.S. Food and Drug Administration; July 1, 2026. Accessed July 2, 2026.