54092-0700-XX Elaprase 6MG/3ML SOLN (SHIRE US INC)
Idursulfase is a purified form of the lysosomal enzyme iduronate-2-sulfatase. It is produced by recombinant DNA technology in a human cell line. This enzyme is required for systemic elimination of the glycosaminoglycans (GAGs) dermatan sulfate and heparan sulfate. If the enzyme is missing or defective, GAGs progressively accumulate in the lysosomes of nearly all cell types, leading to cellular engorgement, organomegaly, tissue destruction and organ system dysfunction. This condition is known as Mucopolysaccharidosis II, MPS II or Hunter syndrome. A rare disease, it is the only known X-linked recessive mucopolysaccharidosis disorder.
MPS II is generally manifest in two forms: Severe disease, affecting two-thirds of those diagnosed, in which death typically occurs in the mid-teenage years due to chronic progressive disease of neurological deterioration and cardiorespiratory failure. The other third have attenuated disease with survival into adulthood, although death frequently occurs between the ages of 20 and 30 years from cardiac or respiratory disease.
While not curative, enzyme replacement therapy with idursulfase can improve quality of life if administered early in the disease state. Additional supportive treatment is generally required for symptomatic control.
Idursulfase for the treatment of Mucopolysaccharidosis II (i.e., MPS II, Hunter syndrome) is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Idursulfase for the treatment of other conditions/diseases is considered investigational.
Idursulfase is considered medically appropriate if ALL of the following criteria are met:
Individual who is 16 months of age or older
Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome) confirmed by ANY ONE of the following:
Deficient iduronate 2-sulfatase (I2S) enzyme activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase
Detection of pathogenic mutations in the IDS gene by molecular genetic testing
Documented baseline Urinary Glycosaminoglycan (uGAG) level
Documented baseline outcomes in ANY ONE of the following:
For individuals 5 years of age or older: 6-minute walk test (6-MWT), percent predicted forced vital capacity (% FVC), joint range of motion, left ventricular hypertrophy, quality of life (CHAQ/HAQ/MPS HAQ)
For individuals less than 5 years of age: spleen volume, liver volume,% FVC, and/or 6-minute walk test
Continued treatment is considered medically appropriate if ALL the following criteria are met:
Individual continues to meet initial approval criteria
Absence of unacceptable toxicity from the agent, including severe hypersensitivity reaction such as anaphylactic and anaphylactoid reactions, antibody development and serious adverse reactions, acute respiratory complications, acute cardiorespiratory failure
Individual has a documented reduction in uGAG levels
Individual has demonstrated a beneficial response to therapy compared to pretreatment baseline in ANY ONE of the following:
Individuals 5 years or greater: stabilization or improvement in percent predicted FVC and/or 6-minute walk test, increased joint range of motion, decreased left ventricular hypertrophy, improved growth, improved quality of life (clinically meaningful change in the CHAQ/HAQ/MPS HAQ disability index)
Individuals < 5 years: spleen volume, and/or liver volume or stabilization/improvement in FVC and/or 6-MWT
|INDICATION(S)||DOSAGE & ADMINISTRATION|
|Mucopolysaccharidosis II (i.e., MPS II, Hunter syndrome)||0.5mg/kg 0.5 mg/kg of body weight administered once weekly as an intravenous infusion|
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed
Refer to DOSAGE LIMITS below
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
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. Idursulfase. Retrieved November 5, 2018 from Lexicomp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2018, September).Idursulfase.Retrieved November 5, 2018 from MICROMEDEX Healthcare Series.
Scarpa, M. (2007, November Updated 2018, October). Mucopolysaccharidosis Type II. In: Adam, M. P., Ardinger, H. H., Pagon, R. A., et al., eds., GeneReviews® (Internet). Seattle (WA): University of Washington, Seattle; 1993-2018. Retrieved November 2, 2018 from https://www.ncbi.nlm.nih.gov/books/NBK1274/.
U.S. Food and Drug Administration. (2013, June). Center for Drug Evaluation and Research. Elaprase® (idursulfase). Retrieved November 5, 2018 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2013/125151s0184lbl.pdf.
ORIGINAL EFFECTIVE DATE: 12/1/201
MOST RECENT REVIEW DATE: 4/9/2019
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; 1 billable unit = 1 mg