Criteria
Coverage is subject to the specific terms of the member's benefit plan.
Betibeglogene autotemcel (Zynteglo) may be considered medically necessary when
ALL
the following criteria are met:
-
The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational);
and
-
The requested medication must be prescribed by, or in consult with, a hematologist or prescriber specializing in the treatment of beta thalassemia;
and
-
The individual must have a transfusion-dependent beta thalassemia requiring one (1) of the following:
-
At least 100 mL/kg/year of packed red blood cells (pRBCs) in the preceding two (2) years;
or
-
At least eight (8) transfusions of pRBCs per year in the preceding two (2) years;
and
-
Other causes of anemia (e.g., hemolysis, bleeding, recent major surgery, vitamin deficiency, etc.) have been ruled out;
and
-
The individual must not have any of the following:
-
Prior receipt of gene therapy;
and
-
Prior HSCT;
and
-
Severely elevated iron in the heart as evidenced by any of the following:
-
Cardiac T2* less than 10 msec by MRI;
or
-
LVEF less than 45%;
and
-
Advanced liver disease as evidenced by any of the following:
-
AST or ALT less than three (3) times the upper limit of normal;
and
-
Direct bilirubin value greater than 2.5 times the upper limit of normal;
and
-
Liver iron content greater than or equal to 15 mg/g (per MRI) with liver biopsy, VCTE, ELF, or MRE demonstrating bridging fibrosis or cirrhosis;
and
- Clinical justification has been provided explaining why the individual is unable to use exagamglogene autotemcel (Casgevy).
Length of Approval:
One (1) course per lifetime
The use of betibeglogene autotemcel (Zynteglo) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.
Procedure Code
