The FDA has expanded the approval of Vertex Pharmaceuticals’ gene therapy Casgevy (exagamglogene autotemcel) to patients 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassemia. The decision makes Casgevy the first gene therapy approved for children as young as 2 with sickle cell disease.
Casgevy was previously approved for patients 12 years and older with the two inherited blood disorders, according to a July 1 FDA news release. The one-time treatment uses CRISPR/Cas9 gene-editing technology to modify a patient’s own blood stem cells to increase fetal hemoglobin production, helping prevent sickled red blood cells and reducing the need for blood transfusions in patients with transfusion-dependent β thalassemia.
The expanded approval was supported by clinical trial data in children 5 years to less than 12 years of age. In the sickle cell disease trial, all eight efficacy-evaluable patients achieved the primary endpoint of remaining free of severe vaso-occlusive crises for at least 12 consecutive months within the first 24 months after treatment.In the β thalassemia trial, eight of nine efficacy-evaluable patients achieved transfusion independence for 12 consecutive months, with a median duration of 20.1 months.
The FDA granted the supplemental approval 53 days after filing through the Commissioner’s National Priority Voucher pilot program. Casgevy also has Orphan Drug, regenerative medicine advanced therapy and Fast Track designations.
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