Treatment of Gaucher Disease

Policy ID: I-9-021

Section: Injections

Effective Date: July 01, 2018

Revised Date: August 31, 2020

Revision Effective Date: October 01, 2020

Last Reviewed: September 12, 2023

Applies To: Commercial Only

Description

Gaucher disease is a genetic disorder resulting in the malfunction or absence of the enzyme glucocerebrosidase. The deficiency of this enzyme causes accumulation of glucosylceramide in the liver, spleen, bone marrow, and lymph nodes. Treatment for this disease focuses on either replacement of the missing or ineffective enzyme (enzyme replacement therapy [ERT]), or reduction of the substrate upon which it acts (substrate reduction therapy [SRT]).

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

Federal Employee Program members (FEP) should check with their Retail Pharmacy Program to determine if prior approval is required by calling the Retail Pharmacy Program at 1-800-624-5060 (TTY: 1-800-624-5077). FEP members can also obtain the list through the www.fepblue.org website.

Imiglucerase (Cerezyme^®), velaglucerase alfa (VPRIV^®) and taliglucerase alfa (Elelyso^®), may be considered medically necessary for individuals with the following:

Initial Authorization Criteria

A diagnosis of Gaucher disease Type I or Type III as confirmed by EITHER of the following:
- Deficiency in glucocerebrosidase in peripheral blood leukocytes or other nucleated cells (e.g., glucocerebrosidase activity of 0-15% of mean normal); or
- Confirmation of biallelic pathogenic variants in the GBA gene; and
Clinical manifestations of non-neuropathic Gaucher disease from ANY ONE of the following conditions:
- Anemia with hemoglobin of:
  - Less than or equal to 11.5 g/dL for females; or
  - Less than or equal to 12.5 g/dL for males; or
  - Less than or equal to 1 g/dL or more below the lower limit of normal for age and sex; or
- Thrombocytopenia with platelet count less than or equal to 120,000/mm³; or
- Bone disease (e.g., osteonecrosis, osteopenia, secondary pathologic fractures); or
- Clinically significant hepatomegaly (liver size 1.25 or more times normal) or splenomegaly (spleen size five (5) or more times normal); or
- Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia.

Reauthorization Criteria

Continuation of therapy with imiglucerase (Cerezyme), velaglucerase alfa (VPRIV), or taliglucerase alfa (Elelyso) may be considered medically necessary when the following criteria are met:

The individual has one of the above diagnoses; and
The prescriber has provided documentation that the individual has demonstrated a disease stability or beneficial response to therapy from baseline as shown by ANY of the following:
- Reduction in liver volume; or
- Reduction in spleen volume; or
- Increase in hemoglobin levels; or
- Increase in platelet counts.

The use of imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), for any other indication is considered experimental/investigational and, therefore, not covered. Scientific evidence of safety and efficacy has not been proven.

Procedure Codes

J1786

J3060

J3385

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the U.S. Food and Drug Administration (FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines.

Diagnosis Codes

E75.22

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 5-19-2020 Annual review, no clinical content change

Internal Medical Policy Committee 9-21-2020 Revision

Initial criteria updated to include Type III Gaucher disease,
Reauthorization criteria added

Internal Medical Policy Committee 9-21-2021 Annual Review

Internal Medical Policy Committee 9-28-2022 Annual Review - no changes in criteria

Internal Medical Policy Committee 9-12-2023

Annual review, no clinical content change

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving, and the Company reserves the right to review and update medical policy periodically.

Section: Injections

Effective Date: June 01, 2020

Revised Date: May 01, 2020

Last Reviewed: May 09, 2020

Archived Date: September 30, 2020

Applies To: Commercial Only

Description

Gaucher disease is a genetic disorder resulting in the malfunction or absence of the enzyme glucocerebroside. The deficiency of this enzyme causes accumulation of glucosylceramide in the liver, spleen, bone marrow, and lymph nodes. Treatment for this disease focuses on either replacement of the missing or ineffective enzyme (enzyme replacement therapy [ERT]), or reduction of the substrate upon which it acts (substrate reduction therapy [SRT]).

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

Imiglucerase (Cerezyme®), taliglucerase alfa (Elelyso®), and velaglucerase alfa (VPRIV®) are indicated for use as a long-term enzyme replacement therapy for pediatric and adult individuals with a confirmed diagnosis of Type I Gaucher disease.

Imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), are indicated for treatment when ONE or more of the following conditions exists in individuals with confirmed Type I Gaucher disease:

Anemia with hemoglobin of:
- Less than or equal to 11.5 g/dL for females; or
- Less than or equal to 12.5 g/dL for males; or
- Less than or equal to 1.0 g/dL or more below the lower limit of normal for age and sex; or
Thrombocytopenia with platelet count less than or equal to 120,000/mm³; or
Bone disease (e.g., osteonecrosis, osteopenia, secondary pathologic fractures); or
Clinically significant hepatomegaly (liver size 1.25 or more times normal) or splenomegaly (spleen size five (5) or more times normal); or
Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia.

The use of imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), for any other indication, including but not limited to the following, is considered experimental/investigational and, therefore, not covered. Scientific evidence of safety and efficacy has not been proven.

Type II Gaucher disease
Type III Gaucher disease

Procedure Codes

J1786

J3060

J3385

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the U.S. Food and Drug Administration (FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines. Blue Cross Blue Shield of North Dakota may deny, in full or in part, reimbursement for utilization that does not fall within the applicable dosage and/or frequency limits.

Diagnosis Codes

E75.22

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Original Effective Date July 1, 2018

Internal Medical Policy Committee 5-19-2020 Annual review, no clinical content change

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving and the Company reserves the right to review and update medical policy periodically.

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