Criteria
Coverage is subject to the specific terms of the member's benefit plan.
Federal Employee Program members (FEP) should check with their Retail Pharmacy Program to determine if prior approval is required by calling the Retail Pharmacy Program at 1-800-624-5060 (TTY: 1-800-624-5077). FEP members can also obtain the list through the www.fepblue.org website.
Betibeglogene autotemcel (Zynteglo) may be considered medically necessary when the following criteria are met:
- The individual has a diagnosis of transfusion dependent Beta-thalassemia (β-thalassemia major or TDT); and
- ONE of the following:
- The individual is less than 12 years of age AND BOTH of the following:
- If the individual is less than five (5) years of age BOTH of the following:
- The individual weighs greater than or equal to six (6) kg; and
- The prescriber has determined that the individual is able to provide the minimum number of cells; and
- ONE of the following:
- The individual has a history of at least 100 mL/kg/year of packed red blood cells (pRBC) in the previous 12 months; or
- The individual has required greater than or equal to eight (8) pRBC transfusions in the past 12 months; or
- The individual is at least 12 years of age but less than or equal to 50 years of age AND ONE of the following:
- The individual has a history of at least 100 mL/kg/year of packed red blood cells (pRBC) in the past 12 months; or
- The individual has required greater than or equal to eight (8) pRBC transfusions in the past 12 months; and
- The individual is clinically stable and able to undergo a hematopoietic stem cell transplant (HSCT); and
- The individual does NOT have a white blood cell count less than 3 X 10^9/L and/or a platelet count less than 100 X10^9/L; and
- The individual does NOT have evidence of an uncorrected bleeding disorder; and
- ONE of the following:
- The individual does NOT have any prior or current malignancy that required systemic therapy; or
- The individual had adequately treated cone-biopsied in situ carcinoma of the cervix uteri; or
- The individual had an adequately treated basal or squamous cell carcinoma of the skin; and
- The individual does NOT have advanced liver dysfunction as defined by any of the following:
- ALT (alanine transaminase) three (3) times the upper limit of normal; and
- Bilirubin above three (3) times the upper limit of normal; and
- Alkaline phosphatase above three (3) times the upper limit of normal; and
- INR (international normalized ratio) greater than or equal to 1.4; and
- The individual does NOT have a T2* less than 10 ms by magnetic resonance imaging (MRI); and
- The individual does NOT have severe iron overload that in the provider's opinion warrants exclusion; and
- The individual is NOT HIV positive; and
- BOTH of the following:
- The individual has a negative hepatitis B surface antigen (HBsAg); and
- ONE of the following:
- The individual's hepatitis B core antibody (HBcAB) is negative; or
- The individual's HBcAB is positive due to a resolved hepatitis B infection AND the individual's HBV virus DNA is negative; and
- ONE of the following:
- The individual's hepatitis C virus (HCV) antibody is negative; or
- The individual's HCV antibody is positive AND the individual's HCV RNA is negative; and
- The individual does NOT have another active infection; and
- The individual has NOT had previous gene therapy for the requested diagnosis.
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
