Zavesca — Blue Cross Blue Shield of Oklahoma

Initial criteria

• ONE of the following: (A) The requested agent is eligible for continuation of therapy AND the patient has been treated with the requested agent (not samples) within the past 90 days AND is at risk if therapy is changed OR (B) all of the following if for Gaucher disease type 1 (GD1): (1) The patient's age is within FDA labeling for the requested indication OR there is support for use for the patient's age; AND (2) Baseline glucocerebrosidase enzyme activity ≤15% of mean normal in fibroblasts, leukocytes, or other nucleated cells OR genetic analysis confirms two pathogenic alleles in the GBA gene; AND (3) The patient does NOT have neuronopathic symptoms indicative of Gaucher type 2 or type 3 (e.g., bulbar, pyramidal, oculomotor, seizure, dementia, ataxia); AND (4) The patient has at least ONE of the following at baseline: anemia (mean hemoglobin below lower limit normal), thrombocytopenia (<100,000/microliter on ≥2 measurements), hepatomegaly, splenomegaly, growth failure (growth velocity below standard mean for age), or bone disease with other causes ruled out; AND (5) Enzyme replacement therapy (ERT) is NOT a therapeutic option (e.g., allergy, poor venous access, previous ERT failure); OR ALL of the following if for Niemann-Pick type C disease (NPC): (A) Diagnosis of Niemann-Pick type C, (B) Genetic analysis confirms mutation in NPC1 or NPC2 genes, (C) Disease-related neurological symptoms, (D) Patient's age is within Miplyffa FDA labeling for the requested indication.
• If the request is for a brand agent with an available generic equivalent (Zavesca/miglustat), then ONE of the following: (A) The patient is currently being treated with and stable on the brand agent; OR (B) The patient has tried and had inadequate response to the generic; OR (C) Generic discontinued due to lack of efficacy, diminished effect, or adverse event; OR (D) Intolerance or hypersensitivity to the generic not expected with brand; OR (E) FDA labeled contraindication to generic not expected to occur with brand; OR (F) Generic expected to be ineffective, cause adherence barrier, worsen comorbidity, decrease ability to function, or cause harm; OR (G) Brand necessary based on medical necessity; OR (H) Tried another drug in same class with inadequate response or intolerance; OR (I) Support for use of brand agent over generic equivalent.
• Prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist), or has consulted with a specialist.
• The patient will NOT be using the requested agent in combination with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Opfolda) for the requested indication.
• The patient does NOT have any FDA labeled contraindications to the requested agent.

Reauthorization criteria

• Patient previously approved for the requested agent through the plan’s Prior Authorization process.
• Patient has had clinical benefit with the requested agent.
• If request for brand agent with available generic equivalent (Zavesca/miglustat), then ONE of the following: same criteria A–I as initial evaluation for brand over generic.
• Prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist), or has consulted with a specialist.
• Patient will NOT be using the requested agent in combination with another substrate reduction therapy agent for the requested indication.
• Patient does NOT have any FDA labeled contraindications to the requested agent.

Approval duration

12 months