Treatment of Gaucher Disease

Section: Injections
Effective Date: May 15, 2019
Revised Date: May 07, 2019

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.

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 ), 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/mm ; 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

Links