Coverage is subject to the specific terms of the member's benefit plan.
The use of imiglucerase (Cerezyme®), velaglucerase alfa (VPRIV®) and taliglucerase alfa (Elelyso®) may be considered medically necessary when ALL of the following criteria are met:
- The individual must meet criteria as outlined in prescribing information (PI) including recommendations for diagnosis and age; and
- The prescriber must be, or in consultation with a geneticist, an endocrinologist, or a physician who specializes in the treatment of lysosomal storage disorders; and
- The individual must have a diagnosis of Gaucher disease Type I or Type III with the one of the following (as evidenced with submitted documentation):
- Deficiency in beta-glucocerebrosidase enzyme activity in peripheral leukocytes; or
- Genetic testing confirming biallelic pathogenic variants in the GBA1 gene; and
- Individual must be experiencing one or more of the following (as evidenced with submitted documentation):
- Anemia with hemoglobin less than or equal to the laboratory reported low for patient age and gender
- Thrombocytopenia with platelet count less than 100,000/mm³
- Bone disease (T-score below -1.0 [DXA], height SDS <-2.25 with decreased growth velocity, bone crisis)
- Hepatomegaly (liver size 1.25 or more times normal)
- Splenomegaly (spleen size five (5) or more times normal); and
- For imiglucerase (Cerezyme) and velaglucerase alfa (VPRIV), information has been provided to explain why these agents are needed over the preferred agent, taliglucerase alfa (Elelyso).
Initial Authorization: 6 months
Continuation of therapy with imiglucerase (Cerezyme®), velaglucerase alfa (VPRIV®) and taliglucerase alfa (Elelyso®) may be considered medically necessary when ALL of the following are met:
- The prescriber has provided documentation that the individual has demonstrated a disease stability or beneficial response to therapy from baseline as shown by one or more of the following:
- Reduction in liver volume to normal size or by 10%
- Reduction in spleen volume by 15%
- Increase in hemoglobin levels by 1mg/dl
- Increase in platelet levels by 15%
- Increased T-score [DXA] by 0.3, normalized growth velocity, or decrease in bone crisis.
Continuation Authorization: 12 months
The use of imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), for any other indication is considered experimental/investigational and, therefore, not covered. Scientific evidence of safety and efficacy has not been proven.