Aqneursa — CareFirst (Caremark)

Niemann-Pick disease type C

Initial criteria

Member weighs ≥ 15 kg
Member age is between 4 years and 64 years
Member has completed the NPC clinical severity scale (NPCCSS) assessment to establish baseline score
Diagnosis is confirmed by either genetically confirmed variant 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 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 not be used in combination with Miplyffa (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C
Medication prescribed by or in consultation with an endocrinologist or physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders

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)

12 months