Coverage is subject to the specific terms of the member’s benefit plan.
The use of emapalumab-lzsg (Gamifant) may be considered medically necessary when ALL of 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
- Emapalumab-lzsg (Gamifant) must be prescribed by, or in consult with, a hematologist, oncologist, immunologist, or transplant specialist; and
- The individual has refractory, recurrent or progressive disease or intolerance with conventional hemophagocytic lymphohistiocytosis (HLH) therapy (i.e., etoposide + dexamethasone, cyclosporine A, or Anti-thymocyte globulin); and
- The individual must be a candidate for stem cell transplant; and
- Documentation must be submitted confirming the diagnosis, as evidenced by the following:
- Confirmation of a gene mutation known to cause primary HLH (e.g., PRF1, UNC13D, STX11 RAB27A, STXBP2); or
- Confirmation of five (5) of the following clinical characteristics:
- Fever greater than or equal to 101.3oF for over seven days
- Two of the following cytopenias in the peripheral blood:
- Hemoglobin less than 9 g/dL (or less than 10 g/dL in infants less than four (4) weeks of age)
- Platelet count less than 100,000/microL
- ANC less than 1000/microL
- One of the following:
- Hypertriglyceridemia defined as fasting triglycerides greater than or equal to 265 mg/dL (2 mmol/L)
- Hypofibrinogenemia defined as fibrinogen less than or equal to 1.5 g/L
- Hemophagocytosis in bone marrow or spleen or lymph nodes with no evidence of malignancy
- Low or absent natural killer cell activity
- Ferritin greater than or equal to 500 mg/L
- Soluble CD25 (i.e., soluble IL-2 receptor) greater than or equal to 2,400 U/mL; and
- Emapalumab-lzsg (Gamifant) must be administered with dexamethasone as part of the induction or maintenance phase of stem cell transplant, which is to be discontinued at the initiation of conditioning for stem cell transplant.
Initial Authorization: 3 months or up to the hematopoietic stem cell transplantation (HSCT) date
Continuation of therapy with emapalumab-lzsg (Gamifant) may be considered medically necessary when the following is met:
- At least three (3) HLH abnormalities must be improved by at least 50% from baseline.
Continuation Authorization: three (3) months or up to the hematopoietic stem cell transplantation (HSCT) date
The use of emapalumab-lzsg (Gamifant) 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.