54092-0701-XX Vpriv 400 UNIT SOLR (SHIRE US INC.)
Velaglucerase alfa is an enzyme replacement agent. It is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for individuals with type I Gaucher disease. It is produced by gene activation technology in a human fibroblast cell line and has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase.
Gaucher disease generally refers to three forms of lysosomal storage disorders (LSD) resulting from the inherited deficiency of the enzyme beta-glucocerebrosidase which cleaves glucose residue from ceramide. Without the natural enzyme, glucocerebroside accumulates in the body, primarily in circulating phagocytes but also in the central nervous system (CNS). The overload of stored glucocerebroside and the attendant activation of macrophages with secretion of cytokines cause the pathology of Gaucher disease.
Type I Gaucher disease is the chronic, non-neuronopathic form of the disease and accounts for nearly 99% of cases. The glucocerebroside storage is limited to phagocytes throughout the body without brain involvement and can manifest at any age. Type II, or acute neuronopathic Gaucher disease, is the infantile cerebral pattern with progressive CNS involvement and early fatality. Type III is subacute neuronopathic disease, an intermediate form between types I and II, with onset in adolescence or early adulthood.
Velaglucerase alfa for the treatment of Gaucher's disease is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Velaglucerase alfa for the treatment of other conditions/diseases is considered investigational.
Velaglucerase alfa for the treatment of Gaucher disease is considered medically appropriate if ALL of the following criteria are met:
Individual is 4 years of age or older
Documented diagnosis of type I Gaucher disease (non-neuronopathic form)
In individual 18 years of age or older, disease results in a minimum of ANY ONE of the following:
Anemia (hemoglobin less than or equal to 11 g/dL [women] or 12 g/dL [men])
Moderate to severe hepatomegaly (liver size 1.25 or more times normal) or splenomegaly (spleen size 5 or more times normal)
Skeletal disease (e.g. lesions, remodeling defects and/or deformity of long bones, osteopenia/osteoporosis, etc.)
Symptomatic disease (e.g. bone pain, fatigue, dyspnea, angina, abdominal distension, diminished quality of life, etc.)
Thrombocytopenia (platelet count less than or equal to 120,000/mm3)
Used as a single agent
Velaglucerase alfa is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Response to treatment is indicated by ANY ONE of the following as compared to pre-treatment baseline:
Improvement in symptoms (e.g. bone pain, fatigue, dyspnea, angina, abdominal distension, diminished quality of life, etc.)
Reduction in size of liver or spleen
Improvement in hemoglobin/anemia
Improvement in skeletal disease
Improvement in platelet counts
Absence of unacceptable toxicity from the drug including hypersensitivity reactions
DOSAGE & ADMINISTRATION
Type 1 Gaucher Disease
Up to 60 units/kg every other week as a 60-minute IV infusion
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed
Refer to DOSAGE LIMITS below
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan, the express terms of the health plan will govern.
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
Lexicomp Online. (2018). AHFS DI. Velaglucerase Alfa. Retrieved September 10, 2018 from Lexicomp Online with AHFS DI.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2016, August). Velaglucerase alfa. Retrieved September 10, 2018 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2015, May). Center for Drug Evaluation and Research. VPRIV™ (velaglucerase alfa). Retrieved September 10, 2018 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2015/022575s017lbl.pdf.
ORIGINAL EFFECTIVE DATE: 9/12/2010
MOST RECENT REVIEW DATE: 10/9/2018
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
This document has been classified as public information
Maximum billable units per dose and over time by indication as a Medical Benefit