Hematology – Zynteglo™

Covers a one‑time (lifetime) IV dose of Zynteglo for transfusion‑dependent β‑thalassemia patients aged 4–50 who meet strict eligibility; excludes prior HSCT or prior gene therapy, severe iron overload, active infections, prior/current malignancy or familial cancer syndromes, significant renal/pulmonary/hepatic dysfunction, prior Zynteglo, concurrent Reblozyl, and children <4. Approval requires extensive documentation including 2‑year transfusion history (≥100 mL/kg/year or ≥8 transfusions/year), genotype confirmation, Hgb ≥11 g/dL before mobilization and conditioning, negative HTLV/HIV and no active infection, performance status ≥80, WBC ≥3×10^9/L and platelets ≥100×10^9/L, eGFR ≥70 mL/min/1.73 m^2 and DLCO ≥50%, planned G‑CSF+plerixafor mobilization, busulfan myeloablative conditioning with VOD prophylaxis, contraception/pregnancy testing, minimum cell dose ≥5.0×10^6 CD34+ cells/kg, and prescription by a hematologist/stem‑cell transplant specialist.

"Patient will undergo mobilization, apheresis and myeloablative conditioning as part of treatment."