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Opfolda — Blue Cross Blue Shield of Kansas

Niemann-Pick type C disease (NPC)

Initial criteria

  • ONE of the following:
  • A. The requested agent is eligible for continuation of therapy AND ONE of the following:
  • 1. The patient has been treated with the requested agent (starting on samples is not approvable) within the past 90 days OR
  • 2. 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. ALL of the following:
  • 1. The patient has a diagnosis of late-onset Pompe disease confirmed by genetic analysis showing biallelic mutation in the GAA gene OR deficient acid alpha-glucosidase enzyme activity in dried blood spots, leukocytes, skin fibroblasts, and/or skeletal muscle tissue AND
  • 2. The patient is not improving on current enzyme replacement therapy (ERT) AND
  • 3. The patient weighs ≥ 40 kg AND
  • 4. The requested agent will be taken in combination with Pombiliti AND
  • 5. If the patient has an FDA labeled indication, then ONE of the following: the patient’s age is within FDA labeling OR there is support for its use for the patient’s age for the requested indication OR
  • C. The requested agent will be used in combination with Miplyffa (arimoclomol) and ALL of the following:
  • 1. The patient has a diagnosis of Niemann-Pick type C disease (NPC) AND
  • 2. Genetic analysis confirms mutation in the NPC1 or NPC2 genes AND
  • 3. The patient has disease-related neurological symptoms AND
  • 4. The patient's age is within Miplyffa FDA labeling for the requested indication AND
  • The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist), or the prescriber has consulted with a specialist AND
  • The patient will NOT be using the requested agent in combination with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Zavesca) for the requested indication AND
  • 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 AND
  • The patient has had clinical benefit with the requested agent AND
  • The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist), or has consulted with a specialist AND
  • The patient will NOT be using the requested agent in combination with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Zavesca) for the requested indication AND
  • The patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

12 months