00069-0106-XX Elelyso 200 UNIT SOLR (PFIZER U.S.)
Taliglucerase alfa is a recombinant active form of the lysosomal enzyme, β-glucocerebrosidase. It is derived from genetically modified carrot plant root cells. Native human β-glucocerebrosidase is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. Taliglucerase alfa differs from native human β-glucocerebrosidase by two amino acids on the N terminal and up to seven amino acids at the C terminal yet it effectively functions in the necessary breakdown of glucocerebroside to glucose and ceramide’
Gaucher disease is a chronic congenital disorder of lipid metabolism caused by deficiency of β-glucocerebrosidase. This leads to accumulation of glycolipids in the liver, spleen and bone marrow. As a result, the individual may develop an enlarged liver and/or spleen, increased skin pigmentation and painful bone lesions. Gaucher disease exists in three clinical subtypes, Type I, Type II and Type III; however enzyme replacement therapy is effective treatment only for confirmed Type I Gaucher disease, the only form determined to be non-neuronopathic.
Taliglucerase alfa for the treatment of Gaucher disease is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Taliglucerase alfa for the treatment of other conditions/diseases is considered investigational.
Taliglucerase alfa is considered medically appropriate if ALL of the following criteria are met:
Disease is confirmed Type I disease (i.e., non-neuronopathic)
Used as a single agent
Age is ANY ONE of the following:
Individual is 4 to 18 years of age
Individual is 18 years of age or older and disease results in 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)
Taliglucerase alfa is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Disease 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 agent such as anaphylaxis, allergic, infusion reactions, etc.
DOSAGE & ADMINISTRATION
Type 1 Gaucher Disease
Up to 60 units/kg every other week as a 60 to 120 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).
Lexi-Comp Online. (2018). AHFS DI. Taliglucerase alfa. Retrieved September 7, 2018 from Lexi-Comp Online with AHFS-DI.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2016, October). Taliglucerase alfa. Retrieved September 7, 2018 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2016, December). Center for Drug Evaluation and Research. Elelyso® (taliglucerase alfa). Retrieved September 7, 2018 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/022458s013lbl.pdf.
ORIGINAL EFFECTIVE DATE: 6/15/2012
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.
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Maximum billable units per dose and over time by indication as a Medical Benefit