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miglustat — Blue Cross Blue Shield of Kansas

Gaucher disease type 1 (GD1)

Initial criteria

  • ONE of the following:
  • A. The requested agent is eligible for continuation of therapy AND ONE of the following: the patient has been treated with the requested agent (not samples) within past 90 days OR the prescriber states the same and patient is at risk if therapy is changed OR
  • B. ONE of the following:
  • 1. The patient has a diagnosis of Gaucher disease type 1 (GD1) AND ALL of the following:
  • A. For FDA labeled indications: patient’s age within labeling OR support for patient’s age AND
  • B. ONE of the following: baseline glucocerebrosidase enzyme activity ≤15% mean normal in fibroblasts/leukocytes/other nucleated cells OR genetic analysis confirms 2 pathogenic alleles in GBA gene AND
  • C. Patient does NOT have neuronopathic symptoms indicative of Gaucher disease type 2 or type 3 (e.g., bulbar signs, strabismus, swallowing difficulty, opisthotonos, seizure, dementia, ataxia) AND
  • D. Patient has ≥1 of: anemia, thrombocytopenia, hepatomegaly, splenomegaly, growth failure, bone disease with other causes ruled out AND
  • E. Enzyme replacement therapy (ERT) is NOT a therapeutic option (e.g., allergy, hypersensitivity, poor venous access, prior ERT failure) OR
  • 2. The requested agent will be used in combination with Miplyffa (arimoclomol) and ALL of the following:
  • A. Diagnosis of Niemann-Pick type C disease AND
  • B. Mutation in NPC1 or NPC2 genes confirmed AND
  • C. Disease-related neurological symptoms present AND
  • D. Patient’s age within Miplyffa FDA labeling for the indication AND
  • If the requested brand agent has a generic equivalent (Zavesca / miglustat), ONE of:
  • A. Patient has intolerance or hypersensitivity to the generic equivalent not expected with brand OR
  • B. Patient has FDA labeled contraindication to generic not expected with brand OR
  • C. Clinical support for using brand over generic AND
  • Prescriber is a specialist (e.g., endocrinologist, geneticist) or has consulted with a specialist AND
  • Patient will NOT use with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Opfolda) for same indication AND
  • Patient does NOT have any FDA labeled contraindications

Reauthorization criteria

  • Patient previously approved through plan’s prior authorization process AND
  • Patient has had clinical benefit with requested agent AND
  • If brand requested (Zavesca/miglustat pair), one of: intolerance/hypersensitivity to generic, contraindication to generic, or clinical support for brand AND
  • Prescriber is a specialist or has consulted with one AND
  • Patient will NOT be using requested agent with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Opfolda) for same indication AND
  • Patient does NOT have any FDA labeled contraindications

Approval duration

12 months