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miglustat — Blue Cross Blue Shield of Montana

Niemann-Pick type C disease (NPC)

Initial criteria

  • ONE of the following:
  • A. The requested agent is eligible for continuation of therapy AND the following:
  • • The prescriber states the patient has been treated with the requested agent (starting on samples is not approvable) within the past 90 days AND is at risk if therapy is changed
  • OR
  • B. ONE of the following apply:
  • 1. The patient has a diagnosis of Gaucher disease type 1 (GD1) AND ALL of the following:
  • • If the patient has an FDA labeled indication, then ONE of the following:
  • – The patient’s age is within FDA labeling for the requested indication for the requested agent OR
  • – There is support for using the requested agent for the patient’s age for the requested indication
  • • ONE of the following:
  • – The patient has baseline glucocerebrosidase enzyme activity ≤15% of mean normal in fibroblasts, leukocytes, or other nucleated cells OR
  • – Genetic analysis confirmed two pathogenic alleles in the glucocerebrosidase (GBA) gene
  • • The patient does NOT have neuronopathic symptoms indicative of Gaucher disease type 2 or 3 (e.g., bulbar signs, pyramidal signs, oculomotor apraxia, tonic-clonic seizures, dementia, ataxia)
  • • The patient has at least ONE of the following at baseline: anemia (Hb below normal range), thrombocytopenia (platelet count <100,000/microliter on at least 2 measurements), hepatomegaly, splenomegaly, growth failure (growth velocity below standard mean for age), or evidence of bone disease with other causes ruled out
  • • Enzyme replacement therapy (ERT) is NOT a therapeutic option (e.g., allergy, hypersensitivity, poor venous access, previous ERT failure)
  • OR
  • 2. The requested agent will be used in combination with Miplyffa (arimoclomol) AND ALL of the following:
  • • The patient has Niemann-Pick type C disease (NPC)
  • • Genetic analysis confirms mutation in NPC1 or NPC2 genes
  • • The patient has disease-related neurological symptoms
  • • The patient’s age is within Miplyffa FDA labeling for the requested indication
  • 2. If the request is for a brand agent with an available generic equivalent (Zavesca/miglustat), then ONE of the following:
  • • The patient is currently being treated with the requested agent and is stable (chart notes required)
  • • The patient has tried and had inadequate response to generic equivalent (chart notes required)
  • • The generic equivalent was discontinued due to lack of efficacy, diminished effect, or adverse event (chart notes required)
  • • The patient has intolerance or hypersensitivity to the generic equivalent not expected with brand agent
  • • The patient has FDA labeled contraindication to the generic equivalent not expected with brand agent
  • • The generic equivalent is expected to be ineffective or cause barriers to care, worsen a comorbid condition, reduce functional ability, or cause harm (chart notes required)
  • • The generic equivalent is not in the best interest of the patient based on medical necessity (chart notes required)
  • • The patient has tried another prescription drug in the same pharmacologic class or mechanism and it was discontinued due to lack of efficacy, diminished effect, or adverse event (chart notes required)
  • • There is support for use of the brand agent over the generic equivalent
  • 3. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist) or has consulted with one
  • 4. The patient will NOT use the requested agent in combination with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Opfolda)
  • 5. The patient has no FDA labeled contraindications to the requested agent

Reauthorization criteria

  • ALL of the following:
  • 1. The patient has been previously approved for the requested agent through the plan’s Prior Authorization process
  • 2. The patient has had clinical benefit with the requested agent
  • 3. If the request is for a brand agent with a generic equivalent (Zavesca/miglustat), ONE of the following:
  • • The patient is currently being treated with the requested agent and is stable (chart notes required)
  • • The patient has tried and had inadequate response to the generic equivalent (chart notes required)
  • • The generic equivalent was discontinued due to lack of efficacy, diminished effect, or adverse event (chart notes required)
  • • The patient has intolerance or hypersensitivity to the generic equivalent not expected with brand agent
  • • The patient has FDA labeled contraindication to the generic equivalent not expected with brand agent
  • • The generic equivalent is expected to be ineffective or cause barriers to care, worsen a comorbid condition, reduce functional ability, or cause harm (chart notes required)
  • • The generic equivalent is not in the best interest of the patient based on medical necessity (chart notes required)
  • • The patient has tried another drug in the same pharmacologic class or mechanism and it was discontinued due to lack of efficacy, diminished effect, or adverse event (chart notes required)
  • • There is support for use of the brand agent over the generic equivalent
  • 4. The prescriber is a specialist in the area of the patient’s diagnosis or has consulted with one
  • 5. The patient will NOT be using the agent with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Zavesca)
  • 6. The patient has no FDA labeled contraindications to the requested agent

Approval duration

12 months