Centene miglustat (Zavesca) Coverage Prior Auth

Coverage Criteria

Initial Therapy - Type 1 Gaucher Disease

Covered when ALL of the following are met:

Type 1 Gaucher Disease Initial: 1) Diagnosis of Type 1 Gaucher disease confirmed by either enzyme assay demonstrating a deficiency in beta-glucocerebrosidase (glucosidase) activity OR DNA testing; 2) Age >= 18 years; 3) Member is symptomatic (e.g., anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly) OR has hypersensitivity/allergy to enzyme replacement therapy (ERT); 4) Failure of two enzyme replacement therapies (e.g., Cerezyme, Elelyso, VPRIV) unless member is unable to take ERT due to intolerance or poor venous access; 5) Zavesca (miglustat) is prescribed as monotherapy; 6) Dose does not exceed 300 mg (3 capsules) per day.

Approval duration: 6 months

Initial Therapy - Niemann-Pick Disease Type C

Covered when ALL of the following are met:

NPC Initial: 1) Diagnosis of Niemann-Pick disease type C (NPC) confirmed by either: a) genetic analysis indicating pathogenic mutation in both alleles of NPC1 or NPC2; OR b) genetic analysis indicating mutation in one allele of NPC1 or NPC2 plus either a positive filipin staining test OR a positive biomarker result (oxysterol, lyso-sphingolipid, bile acid); 2) Prescribed by or in consultation with a geneticist, neurologist, endocrinologist, or metabolic disease specialist; 3) Member presents with at least one neurological sign or symptom of NPC (see Appendix D); 4) Dose does not exceed 600 mg (6 capsules) per day.

Approval duration: 6 months

Continued Therapy - Type 1 Gaucher Disease

Covered when ALL of the following are met:

Type 1 Gaucher Continued: 1) Member is currently receiving medication via Centene benefit or previously met initial approval criteria OR continuity of care applies; 2) Member is responding positively to therapy as evidenced by increased or stabilized platelet count or hemoglobin, reduced or stabilized spleen or liver volume, or decreased bone pain; 3) Zavesca is prescribed as monotherapy; 4) If request is for a dose increase, new dose does not exceed 300 mg (3 capsules) per day.

Approval duration: not explicitly stated in this section

Continued Therapy - Niemann-Pick Disease Type C

Covered when ALL of the following are met:

NPC Continued: 1) Member is currently receiving medication via Centene benefit or previously met initial approval criteria OR continuity of care applies; 2) Member is responding positively as evidenced by improvement or stabilization in a domain affected by NPC (e.g., ambulation, fine motor skills, swallowing, sitting, or speech); 3) If request is for a dose increase, new dose does not exceed 600 mg (6 capsules) per day.

Approval duration: 12 months

Other Indications

Covered when meeting referenced formulary/off-label policies:

Other Indications: If the requested indication (e.g., newly approved indication, age expansion, or dosing regimen) is not yet reflected in this policy, or the drug is not listed for the requested use, follow the relevant formulary, non‑formulary, or off‑label use policies for the applicable line of business (examples: CP.CPA.190, HIM.PA.103, CP.PMN.16, HIM.PA.33, CP.CPA.09, HIM.PA.154, CP.PMN.53).

Requests for use of miglustat for non‑FDA approved indications that are not specifically addressed by this policy are not authorized unless the provider submits sufficient documentation of efficacy and safety in accordance with the applicable off‑label use policies (for example, CP.CPA.09 for commercial, HIM.PA.154 for Health Insurance Marketplace, and CP.PMN.53 for Medicaid) or unless coverage is supported by the member's evidence of coverage documents.

For members covered by Medicaid, state Medicaid coverage provisions take precedence whenever they conflict with this clinical policy. Coverage decisions are subject to the terms, conditions, exclusions, and limitations of the member's evidence of coverage and to applicable state and federal requirements; law and regulation govern in case of any discrepancy.

Initial Therapy

Initial Therapy — Initial therapy criteria by diagnosis

Initial therapy criteria by diagnosis:

Initial - Type 1 Gaucher: See Initial Approval Criteria - Type 1 Gaucher Disease: diagnosis confirmation (enzyme assay or DNA testing), age >= 18 years, symptomatic or unable to receive ERT, failure of two ERTs unless unable to take ERT, monotherapy, dose <= 300 mg/day.

Initial - NPC: See Initial Approval Criteria - NPC: diagnostic confirmation (genetic and/or biomarker/filipin testing as specified), specialist involvement or consultation, presence of at least one neurological sign/symptom, dose <= 600 mg/day.

Continuation Therapy

Continuation Therapy — Continued therapy requirements and response criteria differ by diagnosis

Continued therapy requirements and response criteria differ by diagnosis:

Continued Therapy - Type 1: Member currently receiving medication or previously met initial criteria; responding positively by hematologic parameters (platelet count or hemoglobin), organ volume (spleen or liver) stabilization or reduction, or decreased bone pain; Zavesca as monotherapy; dose limits per indication (<=300 mg/day).

Continued Therapy - NPC: Member currently receiving medication or previously met initial criteria; improvement or stabilization in NPC‑related functional domains (e.g., ambulation, fine motor skills, swallowing, sitting, speech); Zavesca as monotherapy; dose limits per indication (<=600 mg/day).

Approval duration for NPC continuation: 12 months

Provider Actions & Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Requests must meet diagnosis-specific criteria (see Initial Approval Criteria) including diagnostic confirmation and documentation. Coverage is governed by the member's plan terms, conditions, exclusions, and limitations and is subject to applicable state and federal requirements.

Prior authorization must document diagnostic confirmation (e.g., enzyme assay, genetic testing, or positive filipin staining/biomarker where applicable).
Requests should reference any recent policy updates (e.g., added criteria for Niemann-Pick disease type C, 11/26/2024).

Documentation Required

Non‑FDA Indications

Requests for indications that are not FDA-approved and not addressed by this policy require sufficient documentation of efficacy and safety per the applicable off-label use policy and the member's evidence of coverage.

Follow off-label use policies: CP CPA.09 for commercial, HIM.PA.154 for marketplace, CP PMN.53 for Medicaid, as applicable.

Step Therapy Requirements

Step	Requirement
1	Documentation of failure of two enzyme replacement therapies (e.g., Cerezyme, Elelyso, VPRIV) unless member is unable to take ERT due to intolerance or poor venous access

Coding & Clinical Thresholds

Stability thresholds used in some trials

HemoglobinDecrease from baseline (unspecified threshold used in some trials)

Platelet count25% decrease from baseline

Liver volume20% increase from baseline

Spleen volume25% increase from baseline

Quantity Limits

Quantity limits / dosing

Product and strengthZavesca (miglustat) 100 mg capsules

Maximum dose — Type 1 Gaucher disease3 capsules per day (300 mg/day)

Maximum dose — Niemann‑Pick disease type C (NPC)6 capsules per day (600 mg/day)

Definitions

Definition — GDI

AbbreviationGDI = type 1 Gaucher disease

ContextGDI referenced throughout policy for adults with type 1 Gaucher disease

Related clinical featuresAnemia, thrombocytopenia, hepatomegaly, splenomegaly, bone disease

Definition — NPC

AbbreviationNPC = Niemann‑Pick disease type C

Clinical featuresNeurological signs may include hearing loss, vertical supranuclear gaze palsy, dysarthria, ataxia, dystonia, impaired ambulation, dysphagia, seizures, dementia

Diagnostic notes

Background

Miglustat is a glucosylceramide synthase inhibitor approved as monotherapy for adults with mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not appropriate. Type 1 Gaucher disease typically affects visceral organs, bone marrow, and bone, and can present with anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone disease; miglustat provides an oral substrate reduction therapy option for patients who cannot receive enzyme replacement therapy.

OpenPayer

Clinical Policy: Miglustat (Zavesca)

Coverage Criteria

Initial Therapy

Continuation Therapy

Provider Actions & Requirements

Step Therapy Requirements

Coding & Clinical Thresholds

Quantity Limits

Definitions

Background