CurrentCareSourcePolicy N/A

Zavesca (miglustat) — Pharmacy coverage criteria for Gaucher disease type 1

Defines prior authorization, coverage criteria, quantity limits, site of service, and renewal requirements for Zavesca (miglustat) for members with Gaucher disease type 1 under the pharmacy benefit.

Policy Summary

PayerCareSource

PolicyZavesca (miglustat) — Pharmacy coverage criteria for Gaucher disease type 1

Policy CodePolicy N/A

Change TypeRemoved specialty requirementextended approval to 12 months

Effective DateJan 5, 2022

Next Review DateN/A

Key ActionObtain prior authorization and provide a valid NDC; document baseline and objective improvement for reauthorization to receive 12‑month approvals.

SourceLink

POLICY UPDATE CHANGES

Removed prescriber specialty requirement and disease manifestations requirement.

Changed renewal criteria and approval durations from 6 months to 12 months.

Elaborated on diagnostic requirement and removed restriction of ERT within last 6 months.

PA RequiredAuthorization status

HomeSite of service

90 caps/30dQuantity limit

12 monthsApproval duration

>=18Minimum age

Coverage criteria for Zavesca (miglustat)

inv-01: Initial Therapy

Covered when ALL of the following are met for initial authorization:

Initial authorization for GD1: 1. Member is at least 18 years of age; AND 2. Member has a diagnosis of mild to moderate Gaucher disease type 1 (e.g., reduced activity of glucocerebrosidase via enzyme assay (0 to 15% of normal), and/or molecular genetic test documenting mutation of the GBA gene); AND 3. Member is NOT eligible for enzyme replacement therapy (e.g., hypersensitivity, poor venous access, failed after at least 6 months); AND 4. Member does NOT have neurologic symptoms possibly suggestive of type II or III Gaucher disease (e.g., supranuclear gaze palsy, cognitive decline, epilepsy, myoclonus and/or ataxia); AND 5. Dosage limited to 100 mg three times a day (Limit: 90 capsules per 30 days). If all the above requirements are met, medication will be approved for 12 months.age >= 18

Exact diagnostic testing examples provided in policy

inv-02: Continuation Therapy

Covered when ALL of the following reauthorization criteria are met:

Policy Summary

PayerCareSource

PolicyZavesca (miglustat) — Pharmacy coverage criteria for Gaucher disease type 1

Policy CodePolicy N/A

Change TypeRemoved specialty requirementextended approval to 12 months

Effective DateJan 5, 2022

Next Review DateN/A

Key ActionObtain prior authorization and provide a valid NDC; document baseline and objective improvement for reauthorization to receive 12‑month approvals.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Chart notes must document improvement in one or more of the following parameters compared to baseline: hemoglobin level; spleen and/or liver volumes; platelet count; or bone outcomes. If documented, the medication will be approved for an additional 12 months.

documented clinical improvement

Reauthorization depends on objective clinical improvement

CareSource considers use of Zavesca (miglustat) subject to defined coverage criteria. Treatments will be approved only when the specific clinical requirements for initial authorization or reauthorization described in this policy are met; requests that do not meet these criteria may be denied. Prior authorization is required and the medication is billed under the pharmacy benefit with a valid NDC required for claims submission. Approval durations are for 12 months when criteria are satisfied.

Zavesca (miglustat) is not medically necessary for the treatment of conditions that are not listed in this document. Requests for off‑label indications or any use not explicitly covered by the criteria herein should be referred to the plan’s Off‑Label policy and may be denied.

Initial therapy requirements

inv-13: Initial Therapy

Covered when ALL of the following are met for initial authorization:

Initial therapy nodes: Member >=18; diagnosis of mild to moderate Gaucher disease type 1 confirmed by enzyme assay and/or genetic testing (e.g., glucocerebrosidase activity 0–15% of normal or GBA mutation); member is ineligible for enzyme replacement therapy (e.g., hypersensitivity, poor venous access, or failed ERT after at least 6 months); absence of neurologic signs suggestive of type II/III disease (e.g., supranuclear gaze palsy, cognitive decline, epilepsy, myoclonus, ataxia); dosage limited to 100 mg three times daily (Limit: 90 capsules per 30 days).

Approval for 12 months if criteria met.

Reauthorization / continuation requirements

inv-14: Reauthorization

Reauthorization requires objective clinical improvement.

Reauthorization nodes: Chart notes must document improvement in one or more of: hemoglobin level; spleen and/or liver volumes; platelet count; bone outcomes compared to baseline.

Approval for additional 12 months if met.

What providers must do

Prior Authorization

Prior Authorization & Billing

Prior authorization is required. Provide the NDC on the request and ensure the medication is billed through the pharmacy benefit using a valid NDC.

NDC must be provided on prior authorization request
Medication billed via pharmacy benefit

Documentation Required

ERT Ineligibility Requirement

Requests for Zavesca are eligible only when the member is ineligible for enzyme replacement therapy (ERT) due to documented reasons such as hypersensitivity to ERT, poor venous access preventing regular infusions, or failure to respond to ERT after at least 6 months of therapy.

Examples of ERT ineligibility: hypersensitivity, poor venous access, failed ERT (≥6 months)

Documentation Required

Reauthorization Documentation

For reauthorization, submit chart notes that document baseline measures and evidence of improvement in one or more clinical parameters compared to baseline (e.g., hemoglobin level, spleen and/or liver volumes, platelet count, bone outcomes). Approval for continuation is contingent on this documentation.

Chart notes must document baseline and improvement in ≥1 parameter: hemoglobin, spleen/liver volume, platelet count, or bone outcomes
Reauthorizations reviewed for evidence of clinical benefit

Denial Risk

Off‑Label / Not Medically Necessary

Requests for indications not listed in this policy are considered not medically necessary and may be denied. Refer to the Off‑Label policy for requests outside the specified indications (e.g., non‑GD1 uses).

Off‑label or unlisted indications are not covered
See Off‑Label policy for guidance

Billing, dosage limits, and codes

Pharmacy billingNDC

NDC	Valid NDC required for billing

Dosage limit — Zavesca (miglustat)

Dosing100 mg three times a day

Quantity limitLimit: 90 capsules per 30 days

Approval duration when criteria metApproval: 12 months

Step therapy and quantity limits

Step	Requirement	Notes
1
Member must be ineligible for enzyme replacement therapy
Examples of ERT ineligibility include hypersensitivity to ERT, poor venous access, or failed ERT after at least 6 months. Required for initiation of Zavesca (miglustat).

Quantity limit summary

DrugZavesca (miglustat)

Quantity limit90 capsules per 30 days

PerMonth

Allowed site of service

Note

Home administration allowed

Zavesca (miglustat) may be administered at home; home administration is an allowed site of service.

Site of service allowed: Home

Background on disease and therapy

Gaucher disease type 1 (GD1) is a non‑neuronopathic, inherited lysosomal storage disorder caused by mutations in the GBA gene that result in deficient glucocerebrosidase activity and accumulation of glucosylceramide in macrophages. Clinical manifestations commonly include hepatosplenomegaly, anemia, thrombocytopenia, and skeletal complications. Zavesca (miglustat) is an oral substrate reduction therapy approved for GD1 that reduces synthesis of the accumulating substrate as an alternative to enzyme replacement therapy.

Definitions and terms

Gaucher disease type 1 (GD1) — definition and diagnostic note

DefinitionGaucher disease type 1 (GD1) is the most common form of Gaucher disease that does not affect the central nervous system; caused by GBA gene mutations leading to deficient glucocerebrosidase and accumulation of glucosylceramide.

Diagnostic criteria examplesReduced glucocerebrosidase activity by enzyme assay (approximately 0–15% of normal) and/or molecular genetic testing documenting a GBA mutation.

Clinical featuresTypical manifestations include hepatosplenomegaly, anemia, thrombocytopenia, and skeletal disease (bone marrow, spleen, liver involvement).