Zavesca (miglustat) — Highmark
mild to moderate type 1 Gaucher disease
Preferred products
- generic miglustat
Initial criteria
- age ≥ 18 years
- diagnosis of mild to moderate type 1 Gaucher disease confirmed by deficiency in glucocerebrosidase activity in peripheral leukocytes OR genetic testing confirms mutant alleles
- member exhibits ≥ 1 of the following: hepatomegaly (liver size ≥1.25× normal, i.e., ≥2.5% total body weight) OR splenomegaly (≥0.2% total body weight) OR bone disease (osteonecrosis, osteopenia, secondary pathologic fractures, or bone infarct) OR bone marrow complications defined as anemia (hemoglobin ≤11.5 g/dL for females or ≤12.5 g/dL for males) OR thrombocytopenia (platelet count ≤120,000/mm3) OR symptomatic disease (abdominal/bone pain, fatigue, exertional limitation, weakness, or cachexia)
- therapeutic failure, contraindication, or intolerance to one enzyme replacement therapy (Cerezyme [imiglucerase], Elelyso [taliglucerase alfa], or VPRIV [velaglucerase alfa])
- if request is for brand Zavesca, therapeutic failure or intolerance to generic miglustat
Reauthorization criteria
- positive clinical response to therapy