Pharmacy Prior Authorization Criteria

Miplyffa (arimoclomol)

Indications for Prior Authorization

Miplyffa (arimoclomol)

• For diagnosis of Niemann-Pick disease type C (NPC)
  Indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.

Criteria

Miplyffa

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s)

• Diagnosis of Niemann-Pick disease type C (NPC)
AND
• Diagnosis is confirmed by one of the following:
• Genetically confirmed (deoxyribonucleic acid [DNA] sequence analysis) by mutations in both alleles of NPC1 or NPC2
OR
• Mutation in only one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane triol/oxysterols (>2 x upper limit of normal)
AND
• Patient has at least one neurological symptom of the disease (e.g., hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia)
AND
• Patient is 2 years of age or older
AND
• Requested drug will be used in combination with miglustat
AND
• Requested drug will NOT be used in combination with Aqneursa (levacetylleucine)
AND
• Prescribed by or in consultation with a specialist knowledgeable in the treatment of Niemann-Pick disease type C

Miplyffa

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient demonstrates positive clinical response to therapy (e.g., slowing of disease progression, improvement in neurological symptoms of the disease)
AND
• Requested drug will be used in combination with miglustat
AND
• Requested drug will NOT be used in combination with Aqneursa (levacetylleucine)

P & T Revisions

2024-11-07

References

1. Miplyffa Prescribing Information. Zevra Therapeutics, Inc. FL 34747. September 2024.
2. Mengel E, Patterson MC, Da Riol RM et al. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.
3. FDA Review: Miplyffa. Food and Drug Administration Web Site. 2024. http://www.accessdata.fda.gov. Accessed November 4, 2024

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Revision History

• 2024-11-07: New program

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