CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Miglustat (and Opfolda) coverage for Gaucher disease and Niemann-Pick type C

This pharmacy policy governs authorization, continuation, and quantity limits for miglustat and Opfolda for Medicaid members, including FDA-approved and compendial uses, and applies to Neighborhood Health Plan of Rhode Island Medicaid population.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMiglustat (and Opfolda) coverage for Gaucher disease and Niemann-Pick type C

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateDec 1, 2020

Next Review DateN/A

Key ActionObtain prior authorization with documentation of diagnostic confirmation and prior therapy (e.g., enzyme assay or genetic testing for Gaucher; genetic confirmation or supportive testing for NPC).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

Medicaidscope population

6 monthstypical auth period

3/daymiglustat ql

9/dayOpfolda ql

Coverage Criteria

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.

Initial Therapy — Gaucher disease type 1

Covered when ALL of the following are met

Gaucher initial: Diagnosis of Gaucher disease confirmed by enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity OR by genetic testing with documentation provided

Gaucher prior therapy: Member has documented inadequate response or intolerable adverse events with enzyme replacement therapy (ERT), or ERT is not a therapeutic option (e.g., allergy, hypersensitivity, poor venous access)

Initial Therapy — Niemann-Pick disease type C

Covered when ALL of the following are met

NPC diagnostic: Diagnosis confirmed by either: (a) genetically confirmed variant in both alleles of NPC1 or NPC2; OR (b) mutation in one allele of NPC1 or NPC2 plus positive filipin staining or elevated cholestane-triol level (>2 times the upper limit of normal)

NPC step: If the request is for miglustat 100 mg capsules, documentation of inadequate response, intolerance, or contraindication to Opfolda 65 mg capsules is required

Continuation Therapy — Gaucher disease type 1

Covered when ALL of the following are met for reauthorization

Meets initial criteria: Documentation that the patient continues to meet the criteria for initial approval

Clinical response/tolerability: Documentation that the patient is not experiencing an inadequate response or any intolerable adverse events from therapy

Continuation Therapy — Niemann-Pick disease type C

Covered when ALL of the following are met for reauthorization

Meets initial criteria: Documentation that the patient continues to meet the criteria for initial approval

Clinical response/tolerability: Documentation that the patient is not experiencing an inadequate response or any intolerable adverse events from therapy

All indications not expressly listed as FDA‑approved or as accepted compendial uses (for example, uses beyond Neiman‑Pick disease type C and other compendial entries) are considered experimental/investigational and are not medically necessary.

Uses of miglustat or related agents that fall outside the listed FDA‑approved indications and recognized compendial uses are considered not medically necessary and are not covered.

Initial Therapy Rules

Initial therapy rules — Initial authorizations may be granted for 6 months

Initial authorizations may be granted for 6 months when the criteria below are met.

Gaucher initial: Diagnosis confirmed by enzyme assay demonstrating beta-glucocerebrosidase deficiency OR by genetic testing with documentation provided; and documented inadequate response or intolerable adverse events with enzyme replacement therapy (or ERT is not a therapeutic option)

Initial authorization period: 6 months

NPC initial: Diagnosis for NPC confirmed per policy diagnostic criteria; if requesting miglustat 100 mg capsules, documentation must show inadequate response, intolerance, or contraindication to Opfolda 65 mg capsules

Initial authorization period: 6 months

Reauthorization / Continuation Criteria

Reauthorization — Reauthorization is granted for 6 months

Reauthorization is granted for 6 months when continuation criteria are met.

Continuation criteria: Documentation that the patient continues to meet the criteria for initial approval

Reauthorization period: 6 months

Clinical response/tolerability: Documentation that the patient is not experiencing an inadequate response or any intolerable adverse events from therapy

Reauthorization period: 6 months

Provider Actions and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required for initiation and reauthorization of therapy. Initial approvals and reauthorizations are typically granted for 6 months when documentation meets the diagnostic and prior therapy requirements listed below.

Initial and reauthorization approval duration: 6 months when criteria met
Providers must supply diagnostic confirmation (see diagnostic criteria below) and prior therapy details

Step Therapy

NPC step requirement

For Neiman‑Pick disease type C (NPC), if the request is for miglustat 100 mg capsules, the request must include documentation of an inadequate response, intolerance, or contraindication to Opfolda (miglustat 65 mg capsules).

Affected product example: miglustat 100 mg capsules — step requirement to try Opfolda 65 mg capsules first unless inadequate response/intolerance/contraindication is documented

Documentation Required

Diagnostic and Prior Therapy Documentation / Denial Risks

Providers must supply diagnostic confirmation and prior therapy documentation to support medical necessity. Examples of acceptable diagnostic confirmation include enzyme assay demonstrating deficient enzyme activity or genetic testing results as specified per indication (e.g., enzyme assay or genetic testing for Gaucher disease; genetically confirmed biallelic NPC1 or NPC2 variants or the alternate NPC diagnostic combinations). If required diagnostic testing or prior therapy attempts are not documented, the request may be denied. Requests for indications not listed as FDA‑approved or compendial (see Indications) are considered experimental/investigational and are not covered.

Gaucher disease type 1: diagnosis confirmed by enzyme assay demonstrating deficiency of beta‑glucocerebrosidase or by genetic testing; prior intolerance or inadequate response to enzyme replacement therapy must be documented for coverage.
NPC: diagnosis confirmed by either (a) genetically confirmed variants in both alleles of NPC1 or NPC2, or (b) mutation in one allele plus positive filipin staining or elevated cholestane‑triol (>2x ULN).
Reauthorization: documentation that initial approval criteria continue to be met and that the patient is not experiencing an inadequate response or intolerable adverse events from therapy.

Coding and Definitions

inv-07: Cholestane-triol level

Threshold>2 times the upper limit of normal

ContextUsed as supportive diagnostic criterion for Niemann-Pick disease type C when a single NPC1 or NPC2 allele mutation is identified

ApplicationElevated cholestane-triol (>2x ULN) can substitute for second allele confirmation when combined with positive filipin staining or single-allele mutation

inv-14: ERT — definition for enzyme replacement therapy

DefinitionEnzyme replacement therapy (ERT)

Role in policyERT is the standard treatment option for Gaucher disease type 1; miglustat is indicated when ERT is not a therapeutic option

Examples of ERT limitationsSituations include allergy, hypersensitivity, or poor venous access

inv-15: NPC diagnostic criteria

Genetic confirmation (both alleles)Genetically confirmed variant in both alleles of NPC1 or NPC2

Alternative confirmationMutation in one allele of NPC1 or NPC2 PLUS either positive filipin staining or elevated cholestane-triol (>2x ULN)

UseRequired diagnostic criteria for coverage of therapies for Niemann-Pick disease type C (NPC)

Step	Requirement	Notes
1	Documentation of inadequate response, intolerance, or contraindication to Opfolda (miglustat 65 mg capsules)	Required prior to approval of miglustat 100 mg capsules for treatment of Niemann‑Pick disease type C (NPC)

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMiglustat (and Opfolda) coverage for Gaucher disease and Niemann-Pick type C

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateDec 1, 2020

Next Review DateN/A

SourceLink

On This Page

OpenPayer

Miglustat (and Opfolda) coverage for Gaucher disease and Niemann-Pick type C

Coverage Criteria

Initial Therapy Rules

Reauthorization / Continuation Criteria

Provider Actions and Prior Authorization

Coding and Definitions

Step Therapy Requirements

Quantity Limits

Background