CurrentCareSourcePolicy N/A

Gaucher disease enzyme replacement therapy (Cerezyme)

Defines prior authorization and medical necessity criteria for coverage of Cerezyme (imiglucerase) for Gaucher disease Types 1 and 3, specifying prescriber qualifications, dosing limits, and exclusions for CareSource members in NC.

Policy Summary

PayerCareSource

PolicyGaucher disease enzyme replacement therapy (Cerezyme)

Policy CodePolicy N/A

Change TypeMaterial clinical updates

Effective Date

Next Review DateApr 2, 2025

Key ActionObtain prior authorization and document diagnosis by enzyme assay or molecular genetic testing showing biallelic GBA pathogenic variants; approve for 1 year if criteria met.

SourceLink

POLICY UPDATE CHANGES

Gaucher Disease Type 3 was added as an other use with supportive evidence.

Age threshold of 6ge; 2 years was added as a condition of approval.

For genetic diagnosis, requirement specified as molecular genetic testing documenting biallelic pathogenic variants in the GBA gene.

Concomitant use with other approved therapies for Gaucher disease was added to the conditions not recommended for approval.

60 U/kgmax per-dose Type 1 (IV)

120 U/kgmax dose Type 3 (IV)

1 yearinitial approval

6ge; 2 yrsminimum age

Prior auth

Policy Summary

PayerCareSource

PolicyGaucher disease enzyme replacement therapy (Cerezyme)

Policy CodePolicy N/A

Change TypeMaterial clinical updates

Effective Date

Next Review DateApr 2, 2025

Key ActionObtain prior authorization and document diagnosis by enzyme assay or molecular genetic testing showing biallelic GBA pathogenic variants; approve for 1 year if criteria met.

SourceLink

On This Page

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Recommended Authorization Criteria

FDA‑Approved Indication — Gaucher Disease Type 1

Covered for the FDA‑approved indication when ALL of the following are met:

Type 1 FDA‑approved indication

Age: Patient is ≥ 2 years of age.
Diagnostic confirmation (ONE of)
- Demonstration of deficient B‑glucocerebrosidase activity in leukocytes or fibroblasts.
- Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.
Prescriber: Cerezyme is prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder sub‑specialist, or a physician who specializes in the treatment of lysosomal storage disorders.
Dosing: Each individual dose must not exceed 60 U/kg administered intravenously no more frequently than three times per week.
Approve for 1 year.

(operational note): Prior authorization required; approve for 1 year if criteria met.

Other Uses with Supportive Evidence — Gaucher Disease Type 3

Covered as 'Other Uses with Supportive Evidence' when ALL of the following are met:

Type 3 supportive use

Age: Patient is ≥ 2 years of age.
Diagnostic confirmation (ONE of)
- Demonstration of deficient B‑glucocerebrosidase activity in leukocytes or fibroblasts.
- Molecular genetic testing documenting biallelic pathogenic variants in the glucocerebrosidase (GBA) gene.
Indication-specific use restrictions
- Medication is used for the management of impaired growth, hematologic, or visceral symptoms (e.g., splenomegaly, hepatomegaly, anemia, thrombocytopenia).

Requests for concomitant use of Cerezyme with other therapies approved for Gaucher disease are not recommended for approval. The policy explicitly states that concomitant use with other approved treatments (for example, eliglustat, taliglucerase alfa, velaglucerase alfa, or miglustat) has not been evaluated and therefore will not be recommended for coverage.

Requests for doses outside the dosing established in this policy will be reviewed on a case‑by‑case basis by a clinician (for example, a Medical Director or pharmacist). Such requests are not automatically approved; individualized clinical review is required and routine automatic approval is not provided.

Coverage of Cerezyme is not recommended for circumstances that are not listed in the Recommended Authorization Criteria. The policy indicates that situations outside the specified criteria will not be supported for approval and that criteria will be updated as new published data become available.

Drug Codes and Dose Limits

Drugmixed

Cerezyme (imiglucerase)

Genzyme product name listed as subject of policy

inv-07: Per-dose limit for Type 1

Per-dose limitEach individual dose must not exceed 60 U/kg administered intravenously

Maximum frequencyNo more frequently than three times per week

ContextApplies to FDA‑approved indication for Gaucher Disease Type 1 (approve for 1 year when criteria met)

inv-08: Dose limit for Type 3

Dose limitDose must not exceed 120 U/kg administered intravenously

Maximum frequencyNo more frequently than once every 2 weeks

Context

Authorization, Documentation, and Denial Risks

Prior Authorization

Prior Authorization Required

Prior authorization is required for Cerezyme. Approvals are granted for 1 year when the member meets the applicable criteria and dosing in the policy. Extended approvals may be allowed if the patient continues to meet criteria and dosing. Requests for doses outside the established dosing will be considered on a case-by-case basis by a clinician (e.g., Medical Director or pharmacist). Because of the specialized skills required for evaluation, diagnosis, and monitoring, Cerezyme must be prescribed by or in consultation with a specialist (geneticist, endocrinologist, metabolic disorder subspecialist, or physician who specializes in lysosomal storage disorders). Automation: None.

Prior authorization required
Approval duration: 1 year if criteria met
Dosing exceptions reviewed case-by-case by clinician
Prescriber requirement: specialist or consultation with specialist
Automation: None

Clinical Background

Gaucher disease is an autosomal recessive lysosomal storage disorder caused by deficiency of the lysosomal enzyme B‑glucocerebrosidase, which leads to accumulation of glucosylceramide within macrophages (Gaucher cells) and results in visceral, hematologic, and skeletal manifestations. The disorder is classified into Types 1 (non‑neuronopathic), 2 (acute neuronopathic), and 3 (chronic neuronopathic); Type 1 is the most common and typically presents with splenomegaly, hepatomegaly, anemia, thrombocytopenia, and skeletal complications. Diagnosis is established by either demonstration of deficient enzyme activity in leukocytes or fibroblasts or by molecular genetic testing documenting biallelic pathogenic variants in the GBA gene.

Disease Subtype Definitions

inv-14: Gaucher Disease Type 1

DefinitionNon‑neuronopathic variant of Gaucher disease (Type 1)

Key manifestationsVisceral: splenomegaly and hepatomegaly; Hematologic: anemia and thrombocytopenia; Skeletal: bone disease and related complications

Epidemiology noteType 1 is the most prevalent form, representing the majority of Gaucher disease cases in Western populations

inv-15: Gaucher Disease Type 3

DefinitionChronic neuronopathic form of Gaucher disease (Type 3)

Clinical featuresNeurologic manifestations plus systemic features including hematologic and visceral symptoms (e.g., anemia, thrombocytopenia, splenomegaly, hepatomegaly)