Miplyffa (arimoclomol) — CareFirst (Caremark)

Initial criteria

• Member is age 2 to 19 years
• Member has completed the NPC clinical severity scale (NPCCSS) assessment to establish baseline score
• Member is ambulatory (able to walk independently or with assistance)
• Diagnosis is confirmed by either of the following: genetically confirmed variant in both alleles of NPC1 or NPC2 OR variant in only one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane-triol level (>2 times the upper limit of normal)
• Member has neurological manifestations of disease (e.g., loss of fine motor skills, swallowing, speech, ambulation)
• Requested medication will be used in combination with miglustat
• Requested medication will not be used in combination with Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C
• Prescribed by or in consultation with an endocrinologist or physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders

Reauthorization criteria

• Member meets the criteria for initial approval
• Member is experiencing benefit from therapy (e.g., stabilization or improvement in 5-domain NPCCSS score, fine motor skills, swallowing, speech, ambulation)

Approval duration

12 months