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miglustat — Blue Cross Blue Shield of Illinois

Gaucher disease type 1

Initial criteria

  • ONE of the following:
  • A. The requested agent is eligible for continuation of therapy AND the following: Agents Eligible for Continuation of Therapy: All target agents are eligible for continuation of therapy AND 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:
  • 1. The patient has a diagnosis of Gaucher disease type 1 AND ALL of the following:
  • A. 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 OR There is support for using the requested agent for the patient’s age for the requested indication AND
  • B. ONE of the following: Baseline glucocerebrosidase enzyme activity ≤ 15% of mean normal in fibroblasts, leukocytes, or other nucleated cells OR Genetic analysis confirmed two pathogenic alleles in the GBA gene AND
  • C. The patient has no neuronopathic symptoms (type 2 or 3) AND
  • D. Has ≥1 of: anemia (Hb below LLN by age/sex) OR thrombocytopenia <100,000/μL (≥2 times) OR hepatomegaly OR splenomegaly OR growth failure OR bone disease with other causes ruled out AND
  • E. Enzyme replacement therapy (ERT) is NOT a therapeutic option (due to allergy, hypersensitivity, poor venous access, or previous ERT failure) OR
  • 2. The requested agent will be used in combination with Miplyffa (arimoclomol) AND ALL of: diagnosis of Niemann-Pick type C disease (NPC); genetic mutation in NPC1 or NPC2 genes; disease-related neurological symptoms; patient age within Miplyffa FDA labeling.
  • If the request is for a brand with generic equivalent (Zavesca vs miglustat), ONE of: A. patient currently stable on requested agent; B. inadequate response to generic; C. generic discontinued due to lack of efficacy, diminished effect, or adverse event; D. intolerance/hypersensitivity to generic not expected with brand; E. FDA labeled contraindication to generic not expected with brand; F. generic expected ineffective or causes barrier to adherence, worsens comorbidities, harms function, or causes adverse reaction; G. brand is medically necessary; H. tried another drug in same class with failure or adverse event; I. support for brand over generic.
  • Prescriber is a specialist (e.g., endocrinologist, geneticist) or has consulted with one.
  • Patient will NOT use the agent with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Opfolda).
  • Patient has no FDA labeled contraindications.

Reauthorization criteria

  • The patient has been previously approved for the requested agent through the plan’s PA process.
  • The patient has had clinical benefit with the requested agent.
  • If brand with available generic equivalent (Zavesca vs miglustat), ONE of the same brand-preference criteria (stable on brand, inadequate response to generic, etc.) applies.
  • Prescriber is a specialist (e.g., endocrinologist, geneticist) or has consulted with one.
  • Patient will NOT use with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Zavesca/Opfolda).
  • Patient has no FDA labeled contraindications.

Approval duration

12 months