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Aqneursa (levacetylleucine)United Healthcare

Treatment of neurological manifestations of Niemann-Pick disease type C (NPC)

Preferred products

  • miglustat

Initial criteria

  • Diagnosis of Niemann-Pick disease type C (NPC)
  • Diagnosis has been genetically confirmed by mutation analysis of NPC1 and NPC2 genes
  • Aqneursa is being used to treat neurological manifestations of NPC
  • Aqneursa is prescribed in combination with miglustat OR history of failure, contraindication, or intolerance to miglustat
  • Patient is not receiving Aqneursa in combination with Miplyffa (arimoclomol)
  • Aqneursa is prescribed by or in consultation with a provider with expertise in the treatment of NPC

Reauthorization criteria

  • Documentation of positive clinical response to Aqneursa therapy (e.g., slowed disease progression from baseline based on assessment with NPC–specific scales)
  • Aqneursa is prescribed in combination with miglustat OR history of failure, contraindication, or intolerance to miglustat
  • Patient is not receiving Aqneursa in combination with Miplyffa (arimoclomol)
  • Aqneursa is prescribed by or in consultation with a provider with expertise in the treatment of NPC

Approval duration

12 months