Gaucher Disease Oral Agents (Cerdelga, miglustat, Zavesca, Yargesa)
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Defines recommended prior authorization and coverage criteria for the oral Gaucher disease agents Cerdelga (eliglustat) and miglustat (Zavesca, Yargesa, generics) for adults with Type I Gaucher disease; applies to pharmacy benefit coverage determinations by the payer.
No material clinical or coverage changes in this revision.
Coverage Criteria for Oral Gaucher Therapies
inv-01: Initial coverage criteria for Type I Gaucher disease (adults)
Covered when ALL of the following are met
Prescribing specialist requirement and diagnostic confirmation required.
inv-02: Continuation of therapy
Covered when ALL of the following are met for patients already started on therapy
Response to therapy required for reauthorization.
Use of Cerdelga (eliglustat), miglustat (generic, Zavesca, Yargesa) for Type II or Type III Gaucher disease is not recommended for approval. The Company determined that enzyme replacement therapy and substrate reduction therapy have not been accepted in the medical community in the United States as standard or appropriate means of treatment for these neuronopathic forms.
Coadministration of an oral substrate reduction therapy with an enzyme replacement therapy for Gaucher disease (for example, concurrent use involving Cerdelga with Cerezyme, Elelyso, Vpriv, or other enzyme replacement products) is not recommended for coverage.
inv-14: Adults with Type I Gaucher disease — diagnostic and CYP2D6 requirement
CYP2D6 testing must be via an FDA-cleared assay as stated in policy.
Covered Indications
inv-14: Adults with Type I Gaucher disease — diagnosis must be established and eliglustat requires CYP2D6 metabolizer status
Diagnosis of Type I Gaucher disease must be established and eliglustat requires CYP2D6 testing
Enzyme activity threshold per policy.
FDA-cleared CYP2D6 test result required for eliglustat approval.
Eligibility Requirements
Eligibility for coverage requires meeting all policy criteria for adults with Type I Gaucher disease. Specifically, the member must be 18 years of age or older; the drug (Cerdelga or miglustat products) must be prescribed by or in consultation with an appropriate specialist (a geneticist, endocrinologist, metabolic disorder sub‑specialist, or a physician who specializes in treatment of Gaucher disease); and the diagnosis of Type I Gaucher disease must be confirmed by either glucocerebrosidase enzyme activity < 30% of normal (white blood cell or skin fibroblast assay) or by biallelic mutations in the glucocerebrosidase (GBA) gene. For requests for Cerdelga (eliglustat), an FDA‑cleared CYP2D6 metabolizer status test result is required.
Provider Actions and Documentation Requirements
Prior authorization recommended
Prior authorization is recommended for pharmacy benefit coverage of Cerdelga (eliglustat) and miglustat products; approval is recommended only for requests that meet the policy's coverage criteria and when prescribed by or in consultation with an appropriate specialist.
CYP2D6 testing required for Cerdelga
An FDA-cleared CYP2D6 metabolizer status test result is required when requesting Cerdelga (eliglustat).
- If request is for Cerdelga, member must have had an FDA-cleared test showing the member's CYP2D6 metabolizer status.
Miglustat indication for Type I Gaucher disease (adults)
Miglustat is indicated for adult patients with mild to moderate Type I Gaucher disease when enzyme replacement therapy is not a therapeutic option (for example, allergy, hypersensitivity, or poor venous access).
- Use miglustat only for adults with mild to moderate Type I Gaucher disease when ERT is unsuitable.
Prescriber specialty requirement for initial therapy
Initial prescriptions or consults must be from a geneticist, endocrinologist, metabolic disorder sub-specialist, or a physician who specializes in the treatment of Gaucher disease.
- All approvals for initial therapy require the drug be prescribed by or in consultation with an appropriate specialist.
Be prepared to provide patient records and provider credentials on request
The Company may request additional documentation to support coverage determinations, including patient records, test results, and credentials of the provider ordering or performing the service.
- Documentation requested may include patient records, test results and/or credentials of the provider.
- Additional documentation supporting medical necessity must be made available upon request.
Denial risk for lack of medical necessity or investigational use
The Company may deny reimbursement if it determines the drug or services were not medically necessary, investigational or experimental, not within scope of benefits, or if a pattern of inappropriate or excessive billing is found.
- Ensure documentation supports medical necessity to avoid denial.
- Requests outside the Recommended Authorization Criteria may be subject to case-by-case review and denial.
Not recommended for Type II or Type III Gaucher disease
Requests for Cerdelga or miglustat for Type II or Type III Gaucher disease are listed as conditions not recommended for approval and are not supported by the policy.
- Cerdelga, miglustat, Zavesca and Yargesa are not recommended for Type II or Type III Gaucher disease.
- Coadministration with another enzyme replacement therapy for Gaucher disease is not recommended for coverage.
Definitions and Disease Types
Not Covered
Testing or therapy with Cerdelga, miglustat, Zavesca, or Yargesa for Type II or Type III Gaucher disease is not recommended for approval and therefore is considered not covered under this policy.
Background
Gaucher disease is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme glucocerebrosidase, leading to accumulation of glucocerebroside in liver, spleen, bone marrow and bone. Type I is the non‑neuronopathic, most common form and primarily affects visceral organs and bone; Types II and III are neuronopathic forms with neurological involvement (Type II is acute infantile onset; Type III is chronic childhood‑onset). Oral substrate reduction therapies such as eliglustat (Cerdelga) and miglustat (Zavesca, Yargesa) have specific indications and limitations: approval in this policy is limited to adults with confirmed Type I Gaucher disease, while these agents and enzyme replacement therapies are not recommended for Type II or Type III disease.
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