miglustat — Blue Cross Blue Shield of Texas

Initial criteria

• ONE of the following: (A) Continuation of therapy — prescriber states the patient has been treated with the requested agent within past 90 days and is at risk if therapy is changed OR (B) New therapy meeting diagnosis-based criteria
• For Gaucher disease type 1 (GD1): ALL of the following: (a) Patient age within FDA labeling OR there is support for use for patient's age; AND (b) baseline glucocerebrosidase enzyme activity ≤15% of mean normal OR genetic analysis confirmed two pathogenic alleles in GBA gene; AND (c) absence of neuronopathic symptoms indicative of type 2 or 3; AND (d) baseline anemia, thrombocytopenia, hepatomegaly, splenomegaly, growth failure, or bone disease with other causes ruled out; AND (e) enzyme replacement therapy (ERT) is NOT a therapeutic option due to allergy, hypersensitivity, poor venous access, or previous failure
• For Niemann-Pick type C: ALL of the following when used with Miplyffa (arimoclomol): diagnosis of NPC; genetic analysis confirms mutation in NPC1 or NPC2 genes; disease-related neurological symptoms; patient's age within Miplyffa FDA labeling
• If brand Zavesca is requested when generic equivalent miglustat is available: ONE of the following is met [chart notes required where indicated]: (A) patient is currently treated and stable on brand agent; OR (B) inadequate response to generic; OR (C) generic discontinued due to lack of efficacy/effectiveness or adverse event; OR (D) intolerance/hypersensitivity to generic not expected to occur 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 functional ability, or cause harm; OR (G) brand use is based on medical necessity in best interest of patient; OR (H) patient tried another drug in same class with discontinuation due to lack of efficacy/effectiveness or adverse event; OR (I) there is support for use of brand over generic
• Prescriber is a specialist in endocrinology, genetics, or has consulted with a specialist
• Patient will NOT use requested agent in combination with another substrate reduction therapy agent (e.g., Cerdelga, eliglustat, Opfolda, Zavesca)
• Patient has no FDA-labeled contraindications to requested agent

Reauthorization criteria

• Patient previously approved through plan’s Prior Authorization process
• Patient has had clinical benefit with requested agent
• If request is for brand Zavesca (when generic equivalent miglustat available): ONE of the following (chart notes required as applicable): currently treated/stable; inadequate response to generic; generic discontinued due to lack of efficacy/effectiveness or adverse event; intolerance/hypersensitivity to generic not expected with brand; FDA-labeled contraindication to generic not expected with brand; generic expected to be ineffective or cause harm/adherence issues; medical necessity best interest; or prior drug in same class discontinued due to lack of efficacy/adverse event; or support for brand over generic
• Prescriber is a specialist in endocrinology, genetics, or has consulted with a specialist
• Patient will NOT use requested agent in combination with another substrate reduction therapy agent
• Patient has no FDA-labeled contraindications to requested agent

Approval duration

12 months