Miplyffa™

Drug - Miplyffa™ (arimoclomol) [Zevra Therapeutics]

June 2025

Therapeutic Area - Niemann-Pick disease type C (NPC)

Initial approval criteria

  • Patient is at least 2 years of age; AND
  • Patient has a diagnosis of Niemann-Pick disease Type C; AND
  • Genetic analysis confirms mutation in the NPC1 or NPC2 genes; AND
  • Patient has disease-related neurological symptoms; AND
  • Miplyffa will be used in combination with miglustat; AND
  • Patient does not have any FDA labeled contraindications to Miplyffa; AND
  • Prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist, neurologist) or prescriber has consulted with a specialist in the area of patient’s diagnosis.
  • Initial approval is for 6 months

Renewal criteria

  • Patient has had clinical benefit with Miplyffa AND
  • Patient does not have any FDA labeled contraindications to Miplyffa; AND
  • Prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist, neurologist) or prescriber has consulted with a specialist in the area of patient’s diagnosis.
  • Renewal approval is for 12 months

Quantity limits

  • 90 capsules per 30 days.  Maximum 124 mg three times a day.
  • Patient’s weight (in kg) must be submitted at time of request.

Questions?

Provider Call Center (844) 575-7887