Zavesca — Blue Cross Blue Shield of New Mexico

Initial criteria

• ONE of the following: (A) The requested agent is eligible for continuation of therapy AND prescriber states the patient has been treated with the requested agent within the past 90 days and is at risk if therapy changed OR (B) Diagnostic and clinical criteria for disease
• If diagnosis = Gaucher disease type 1 (GD1): ALL of the following: (A) If the patient has an FDA labeled indication, then ONE of the following: patient’s age is within FDA labeling OR there is support for requested agent use for patient’s age; AND (B) ONE of the following: baseline glucocerebrosidase enzyme activity ≤15% of mean normal in fibroblasts, leukocytes, or other nucleated cells OR genetic analysis confirmed two pathogenic alleles in the glucocerebrosidase (GBA) gene; AND (C) Patient has no neuronopathic symptoms indicative of Gaucher disease type 2 or 3; AND (D) Patient has ≥1 of: anemia (mean Hb below normal lab range), thrombocytopenia (<100,000/μL on ≥2 tests), hepatomegaly, splenomegaly, growth failure (growth velocity below standard mean for age), or bone disease with other causes ruled out; AND (E) Enzyme replacement therapy (ERT) is NOT a therapeutic option (e.g., due to allergy, hypersensitivity, poor venous access, prior ERT failure)
• If indication = Niemann-Pick type C disease (NPC): ALL of: diagnosis confirmed, genetic analysis shows NPC1 or NPC2 mutation, presence of neurological symptoms, patient’s age within Miplyffa FDA labeling, and agent used in combination with Miplyffa (arimoclomol)
• If the request is for brand agent Zavesca with available generic equivalent miglustat: ONE of the following: (A) Patient currently stable on requested brand, (B) tried and had inadequate response to generic, (C) generic discontinued due to lack of efficacy/effectiveness, diminished effect, or adverse event, (D) intolerance or hypersensitivity to generic not expected with brand, (E) FDA labeled contraindication to generic not expected with brand, (F) generic expected to be ineffective or cause adherence barrier, worsen comorbid condition, decrease functional ability, or cause harm, (G) brand is medically necessary, (H) tried another drug in same class discontinued due to ineffectiveness/adverse event, or (I) support for brand use over generic equivalent
• Prescriber is a specialist (endocrinologist, geneticist) or has consulted with one
• Patient will NOT use in combination with another substrate reduction therapy (e.g., Cerdelga, eliglustat, Opfolda)
• Patient does NOT have any FDA labeled contraindications to the requested agent

Reauthorization criteria

• Patient previously approved for the requested agent through plan’s prior authorization process
• Patient has had clinical benefit with the requested agent
• If brand requested with generic available (Zavesca vs miglustat): same brand-vs-generic criteria as initial
• Prescriber is specialist or has consulted specialist
• Patient not using another substrate reduction therapy concurrently
• Patient does not have any FDA labeled contraindication to the requested agent

Approval duration

12 months