CurrentCignaPolicy N/A

Gaucher Disease - Substrate Reduction Therapy Preferred Specialty Management Policy

Defines prior authorization, preferred product steering, and exception criteria for substrate reduction therapies (Cerdelga, Zavesca, Yargesa/generic miglustat) for treatment of Gaucher disease type 1 for Cigna-administered plans.

Policy Summary

PayerCigna

PolicyGaucher Disease - Substrate Reduction Therapy Preferred Specialty Management Policy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateOct 22, 2025

Key ActionObtain prior authorization and documentation of trials of preferred products when requesting non-preferred Zavesca.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

3drugs referenced

1 yearstandard approval duration

NCapplicable state

Coverage Criteria

Non-Preferred Product Exception Criteria (Zavesca)

Non-Preferred Product exception criteria for Zavesca

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Zavesca Exception Criteria Group 1 (Gaucher Disease Type I)

1Ai: Patient meets the standard Gaucher Disease Substrate Reduction Therapy - Miglustat Prior Authorization criteria
1Aii: Patient has tried Cerdelga (eliglustat) [documentation required]
Documentation may include chart notes, prescription claims records, or prescription receipts and must include patient-specific identifying information.
1Aiii: Patient meets BOTH: (a) has tried ONE of Yargesa or generic miglustat capsules [documentation required]; AND (b) brand Zavesca is requested due to a formulation difference in inactive ingredient(s) which, per the prescriber, has or would result in a significant allergy or serious adverse reaction

Zavesca Exception Criteria Group 1B: For a patient who meets 1Ai but does not meet 1Aii and 1Aiii, offer to review for one of the Preferred Products using the standard Cerdelga or Miglustat Prior Authorization criteria.

Zavesca Exception Criteria Group 2 (Other Conditions)

2Ai: Patient meets the standard Miglustat Prior Authorization criteria
2Aii: Patient meets BOTH: (a) has tried ONE of Yargesa or generic miglustat capsules [documentation required]; AND (b) brand Zavesca is requested due to a formulation difference in inactive ingredient(s) which, per the prescriber, has or would result in a significant allergy or serious adverse reaction

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for all medications (preferred and non-preferred). Requests for non-preferred products (including Zavesca) will be reviewed under the non-preferred product exception criteria and the applicable prior authorization criteria. All approvals are time-limited as noted in the policy.

Prior authorization required for all medications (Preferred and Non-Preferred).
Non-preferred requests reviewed using exception criteria; approvals are time-limited.

Step Therapy

Preferred-before-Nonpreferred Requirement

The program requires trial of the Preferred Product(s) before approving a Non‑Preferred product. Patients who meet prior authorization criteria for a non-preferred product but have not tried preferred options will be offered review for a preferred product (e.g., Cerdelga or miglustat/Yargesa per applicable PA criteria).

Preferred-before-nonpreferred requirement: patient must try Preferred Product(s) prior to approval of Non-Preferred.
If patient has not tried Preferred Product(s), offer review for Preferred Product using the standard PA criteria.

Documentation Required

Documentation Requirements

Documentation is required for requests involving Cerdelga, Yargesa, generic miglustat, and Zavesca where noted. Acceptable documentation may include chart notes, prescription claims records, prescription receipts, and must include patient-specific identifying information. Documentation should corroborate prior trials of preferred products when required by the exception criteria.

Documentation may include chart notes, prescription claims records, and prescription receipts.
All documentation must include patient-specific identifying information.
Documentation required to verify trials of Cerdelga, Yargesa, or generic miglustat as specified in criteria.

Denial Risk

Coding Noncompliance May Cause Denial

Claims submitted without covered diagnosis or procedure codes consistent with the applicable coverage policy may be denied as not covered. Providers must bill using the most appropriate codes as of the date of submission; coding noncompliance can result in claim denial.

Claims lacking covered diagnosis/procedure codes per the coverage policy will be denied as not covered.
Use the most appropriate codes as of the submission date to avoid denial.

Definitions

Cerdelga (eliglustat) — substrate reduction therapy

Drug and classCerdelga (eliglustat) — substrate reduction therapy for Gaucher disease type 1.

IndicationIndicated for long-term treatment of adult patients with Gaucher disease type 1.

CYP2D6 guidanceCerdelga is indicated specifically for patients who are CYP2D6 extensive, intermediate, or poor metabolizers as detected by an FDA-cleared test.

Related productsUsed within the same substrate reduction therapy class as miglustat formulations (Zavesca, Yargesa).

Miglustat (Zavesca, Yargesa/generic) — monotherapy indication

Drug namesMiglustat formulations: Zavesca (brand), Yargesa (branded generic), and generic miglustat.

IndicationMiglustat capsules are indicated as monotherapy for adult patients with mild to moderate Gaucher disease type 1 when enzyme replacement therapy is not a therapeutic option.

Clinical scenarios for useUsed when enzyme replacement therapy is not possible due to reasons such as allergy, hypersensitivity, or poor venous access.

Formulation noteYargesa is a branded generic supplied in the same dosage strength as Zavesca.

Initial Therapy Criteria

Preferred Product Trial Requirement

Preferred product use before non-preferred

Preferred product trial: Patient should try Preferred Product(s) prior to approval of Non-Preferred Zavesca; requests for Non-Preferred will be reviewed using the exception criteria. Patients meeting prior authorization criteria for a Non-Preferred Product who have not tried Preferred Product(s) will be offered a review for a Preferred Product.

Documentation may be required to demonstrate trials of preferred products.

Step Therapy Requirements

Step	Requirement
1	Trial of Cerdelga (eliglustat capsules) [documentation required] prior to approval of non-preferred Zavesca; documentation may include chart notes, prescription claims records, or prescription receipts.
1	Trial of ONE of Yargesa or generic miglustat capsules [documentation required] is required in many exception pathways before brand Zavesca may be approved when requested for formulation-related allergy or serious adverse reaction.
1	If patient meets standard Miglustat or Cerdelga prior authorization criteria but has not tried the Preferred Products, offer review for a Preferred Product using the applicable prior authorization criteria.

OpenPayer

Gaucher Disease - Substrate Reduction Therapy Preferred Specialty Management Policy

Coverage Criteria

Coding and Testing

Provider Actions and Requirements

Background

Definitions

Initial Therapy Criteria

Continuation and Approval Duration

Step Therapy Requirements