Cerdelga (eliglustat) — Highmark
type 1 Gaucher disease
Initial criteria
- age ≥ 18 years
- diagnosis of 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% of 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)
- documentation of an FDA-cleared test confirming CYP2D6 extensive, intermediate, or poor metabolizer genotype
- appropriate quantity being utilized based on CYP2D6 metabolizer status
Reauthorization criteria
- positive clinical response to therapy