Miplyffa (arimoclomol) — United Healthcare
Niemann-Pick disease type C (NPC)
Initial criteria
- Diagnosis of Niemann-Pick disease type C (NPC)
- Diagnosis has been genetically confirmed by mutation analysis of NPC1 and NPC2 genes
- Miplyffa is being used to treat neurological manifestations of NPC
- Miplyffa is prescribed in combination with miglustat
- Patient is not receiving Miplyffa in combination with Aqneursa (levacetylleucine)
- Miplyffa is prescribed by or in consultation with a provider with expertise in the treatment of NPC
Reauthorization criteria
- Documentation of positive clinical response to Miplyffa therapy (e.g., slowed disease progression from baseline based on assessment with NPC–specific scales)
- Miplyffa continues to be prescribed in combination with miglustat
- Patient is not receiving Miplyffa in combination with Aqneursa (levacetylleucine)
- Miplyffa is prescribed by or in consultation with a provider with expertise in the treatment of NPC
Approval duration
12 months