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miglustat — Blue Cross Blue Shield of Alabama

Gaucher disease type 1 (GD1)

Initial criteria

  • Prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist) or has consulted with a specialist in the area of the patient’s diagnosis
  • Patient will NOT be using the requested agent in combination with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Opfolda) for the requested indication
  • Patient does NOT have any FDA labeled contraindications to the requested agent
  • ONE of the following:
  • • Patient has been treated with the requested agent within the past 90 days (starting on samples is not approvable)
  • • Prescriber states the patient has been treated with the requested agent within the past 90 days and is at risk if therapy is changed
  • OR
  • Patient has a diagnosis of Gaucher disease type 1 (GD1) AND ALL of the following:
  • • Patient’s age is within FDA labeling for the requested indication OR there is support for using the requested agent for the patient’s age
  • • Baseline glucocerebrosidase enzyme activity ≤15% of mean normal OR genetic analysis confirmed two pathogenic GBA alleles
  • • No neuronopathic symptoms indicative of Gaucher disease type 2 or 3
  • • At least ONE baseline clinical presentation: anemia, thrombocytopenia (platelet <100,000/μL on at least 2 measurements), hepatomegaly, splenomegaly, growth failure, or bone disease with other causes ruled out
  • • Enzyme replacement therapy (ERT) is NOT a therapeutic option (e.g., due to allergy, hypersensitivity, poor venous access, previous ERT failure)
  • OR
  • Requested agent will be used in combination with Miplyffa (arimoclomol) AND ALL of the following:
  • • Diagnosis of Niemann-Pick disease type C (NPC)
  • • Genetic analysis confirms mutation in NPC1 or NPC2 genes
  • • Patient has disease-related neurological symptoms
  • • Patient’s age is within Miplyffa FDA labeling for the requested indication
  • Brand/generic consideration (if requesting Zavesca when generic miglustat is available): ONE of the following:
  • • Intolerance or hypersensitivity to the generic equivalent not expected to occur with the brand agent
  • • FDA labeled contraindication to the generic equivalent not expected to occur with the brand agent
  • • Support for the use of the brand agent over the generic equivalent

Reauthorization criteria

  • Patient has been previously approved for the requested agent through the plan’s Prior Authorization process
  • Patient has had clinical benefit with the requested agent
  • Brand/generic consideration (if requesting Zavesca when generic miglustat is available): ONE of the following:
  • • Intolerance or hypersensitivity to the generic equivalent not expected to occur with the brand agent
  • • FDA labeled contraindication to the generic equivalent not expected to occur with the brand agent
  • • Support for the use of the brand agent over the generic equivalent
  • Prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist) or has consulted with a specialist in the area of the patient’s diagnosis
  • Patient will NOT be using the requested agent in combination with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Opfolda) for the requested indication
  • Patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

12 months