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Opfolda — Blue Cross Blue Shield of Alabama

Niemann-Pick type C disease (NPC)

Initial criteria

  • ONE of the following:
  • - The requested agent is eligible for continuation of therapy AND ONE of the following:
  • • The patient has been treated with the requested agent (starting on samples is not approvable) within the past 90 days OR
  • • 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
  • ALL of the following:
  • - For late-onset Pompe disease:
  • • Diagnosis confirmed by genetic analysis showing biallelic pathogenic variants in GAA gene OR deficient acid alpha-glucosidase enzyme activity in dried blood spots, leukocytes, skin fibroblasts, and/or skeletal muscle tissue
  • • Patient weighs ≥ 40 kg
  • • Patient is not improving on current enzyme replacement therapy (ERT)
  • • Requested agent will be taken in combination with Pombiliti
  • • 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
  • - For Niemann-Pick type C disease (used in combination with Miplyffa):
  • • The patient has a diagnosis of Niemann-Pick type C disease
  • • 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
  • - The prescriber is a specialist (e.g., endocrinologist, geneticist) or has consulted with one
  • - The patient will NOT be using the requested agent in combination with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Zavesca)
  • - The patient does NOT have any FDA labeled contraindications to the requested agent

Reauthorization criteria

  • - The patient has been previously approved for the requested agent through the plan’s Prior Authorization process
  • - The patient has had clinical benefit with the requested agent

Approval duration

12 months