Anthem Blue Cross Connecticut Commercial Clinical Policy

Anthem Blue Cross Connecticut MED.00140 Gene Therapy for Beta Thalassemia Form


Betibeglogene autotemcel infusion for Beta Thalassemia

Indications

(661396) Does the patient have a diagnosis of beta thalassemia? 
(661397) Is the patient transfusion-dependent, needing at least 8 transfusions or at least 100 ml/kg of body weight of packed red cells per year in the previous 2 years? 
(661398) Is the patient a candidate for an allogeneic hematopoietic cell transplantation and ineligible due to the absence of a suitable donor (HLA 8/8 or 7/8 unrelated donor or matched related donor)? 
(661399) Does the patient have no evidence of severe iron overload as indicated by T2*-weighted MRI measurements of myocardial iron greater than 10 msec? 
(661400) Does the patient have no serious concomitant illness (e.g., advanced liver disease, uncorrected bleeding disorder, current malignancy, myeloproliferative and/or immunodeficiency disorder, uncontrolled seizure disorder)? 

Contraindications

(661401) Are the medically necessary criteria not met? 
(661402) Is Betibeglogene autotemcel being considered for indications other than those specified in the medically necessary criteria? 
Effective Date

01/18/2024

Last Reviewed

01/16/2024

Original Document

  Reference



This document addresses gene therapy for beta thalassemia, a genetic disease that involves mutations in the human beta-globin (HBB) gene. These mutations reduce an affected individual’s ability to produce hemoglobin and lead to a shortage of mature red blood cells and a lack of sufficient oxygen. Lentiviral vector gene therapy involves using a modified lentivirus to deliver a functional copy of the beta-globin gene to the patient's cells.

One gene therapy product for beta thalassemia, betibeglogene autotemcel (Zynteglo®), has been approved by the Food and Drug Administration (FDA). Zynteglo is an autologous hematopoietic stem cell-based gene therapy that requires patients to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for Zynteglo manufacturing, as well as administration of full myeloablative conditioning before infusion of Zynteglo.

Note: Please see the following related documents for additional information:

  • CG-MED-68 Therapeutic Apheresis
  • CG-MED-90 Chelation Therapy
  • MED.00146 Gene Therapy for Sickle Cell Disease
  • TRANS.00029 Hematopoietic Stem Cell Transplantation for Genetic Diseases and Aplastic Anemias

Position Statement

Medically Necessary:

A one-time infusion of betibeglogene autotemcel is considered medically necessary in individuals when all of the following criteria are met:

  1. Diagnosis of beta thalassemia; and
  2. Transfusion-dependent disease (that is, needing at least 8 transfusions or at least 100 ml per kilogram of body weight of packed red cells per year in the previous 2 years); and
  3. The individual is a candidate for an allogeneic hematopoietic cell transplantation, but ineligible due the absence of a donor*; and
  4. Has no evidence of severe iron overload (for example, T2*-weighted magnetic resonance imaging [MRI] measurements of myocardial iron greater than 10 msec); and
  5. No serious concomitant illness (for example, advanced liver disease, uncorrected bleeding disorder, current malignancy, myeloproliferative and/or immunodeficiency disorder, uncontrolled seizure disorder).

*Documentation that a suitable donor has not been identified, for example, a matched related donor or matched (HLA 8/8 or 7/8) unrelated donor.

Investigational and Not Medically Necessary:

Betibeglogene autotemcel is considered investigational and not medically necessary when the criteria above are not met, and for all other indications.