Aqneursa (levacetylleucine)
Indications for Prior Authorization
Aqneursa (levacetylleucine)
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For diagnosis of Niemann-Pick disease type C (NPC)
Indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing greater than or equal to 15kg.
Criteria
Aqneursa
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)
- 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 weighs greater than or equal to 15kg AND
- Requested drug will not be used in combination with Miplyffa (arimoclomol) AND
- Prescribed by or in consultation with a specialist knowledgeable in the treatment of Niemann-Pick disease type C
Aqneursa
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 not be used in combination with Miplyffa (arimoclomol)
P & T Revisions
2024-11-07
References
- Aqneursa Prescribing Information. IntraBio Inc. Austin TX 78701. September 2024.
- Bremova-Ertl T, Ramaswami U, Brands M, et al. Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C. N Engl J Med. 2024 Feb 1;390(5):421-431. doi: 10.1056/NEJMoa2310151. PMID: 38294974.
- FDA Review: Aqneursa. Food and Drug Administration Web Site. 2024. http://www.accessdata.fda.gov. Accessed November 4, 2024
Revision History
- 2024-11-07: New program
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