Gene Therapies for Thalassemia

This policy covers gene therapies for transfusion‑dependent β‑thalassemia—specifically betibeglogene autotemcel (Zynteglo™) and exagamglogene autotemcel (Casgevy®). Coverage is limited to patients with documented transfusion‑dependent β‑thalassemia (typically β‑thalassemia major, occasionally intermedia) who meet specified transfusion-history thresholds (≥100 mL/kg/year or ≥8 transfusions/year for each of the prior 2 years), are eligible for HSCT-style conditioning with adequate performance status and organ function, and satisfy other criteria; exclusions include prior allogeneic transplant or prior gene therapy, severe iron overload, repeat dosing (generally one lifetime administration), and benefit‑plan/compendia-based limits.

"Drug therapy that is proven effective for the relevant diagnosis or procedure."

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