Miplyffa (arimoclomol capsules - Zevra) — Cigna

Niemann-Pick disease type C

Initial criteria

Patient is age ≥ 2 years; AND
Patient has one or more neurological symptom(s) of Niemann-Pick disease type C; AND
Patient can walk independently or with assistance; AND
Diagnosis is established by a genetic test showing biallelic pathogenic variants in either the NPC1 gene or NPC2 gene; AND
Patient does NOT have adult-onset Niemann-Pick disease type C (defined as age of first neurological symptom occurring > 15 years of age); AND
ONE of the following: (a) The medication will be taken in combination with miglustat; OR (b) According to the prescriber, patient is unable to take miglustat; AND
Medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician who specializes in the treatment of Niemann-Pick disease type C or related disorders.

Patient does NOT have adult-onset Niemann-Pick disease type C (defined as age of first neurological symptom occurring > 15 years of age); AND
ONE of the following: (a) The medication will be taken in combination with miglustat; OR (b) According to the prescriber, patient is unable to take miglustat; AND
According to the prescriber, patient has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement; AND
Medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician who specializes in the treatment of Niemann-Pick disease type C or related disorders.

1 year