Miplyffa (arimoclomal) — Highmark
Niemann-Pick Disease Type C with neurological manifestations
Initial criteria
- Diagnosis of Niemann-Pick Disease Type C (ICD-10: E75.242) confirmed by mutations in the NPC1 or NPC2 genes
- Member has neurological symptoms of Niemann-Pick Disease Type C (e.g., ataxia, dysarthria, dysphagia, cognitive impairment, seizures)
- Member is age ≥ 2 years
- Medication will be used in combination with miglustat
Reauthorization criteria
- Prescriber attests that the member has experienced positive clinical response to therapy
Approval duration
12 months