68135-0100-XX Vimizim 5 MG/5ML SOLN (BIOMARIN PHARMACEUTICALS)
Elosulfase alfa is a purified human enzyme produced by recombinant DNA technology. It is used for enzyme replacement therapy for individuals with mucopolysaccharidosis IVA (MPS IVA, Morquio A syndrome). Mucopolysaccharidoses are a group of lysosomal storage disorders caused by the deficiency of specific enzymes required for the catabolism of glycosaminoglycans (GAG). The enzyme deficient or absent in MPS IVA is N-acetylgalactosamine-6-sufatase leading to accumulation of the GAG substrates keratan sulfate (KS) and chondroitin-6-sulfate (C6S) throughout the body, eventually leading to widespread cellular, tissue and organ dysfunction.
Elosulfase alfa for the treatment of mucopolysaccharidosis IVA is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Elosulfase alfa for the treatment of other conditions/diseases is considered investigational.
Elosulfase alfa is considered medically appropriate if ALL of the following criteria are met:
Individual is 5 years of age or older
Documented diagnosis of mucopolysaccharidosis IVA (MP IVA, Morquio A syndrome) with biochemical/genetic confirmation by ANY ONE of the following:
Absence or marked reduction in N-acetylgalactosamine 6-sulfatase (GALNS) enzyme activity
Sequence analysis and/or deletion/duplication analysis of the GALNS gene for biallelic mutation
Documented baseline value for one or more of the following: endurance tests ( e.g.,six minute walk test (6-MWT) or timed 25-foot walk test (T25FW)), pulmonary function tests (e.g., FVC), etc.
Product is prepared and administered under supervision of a healthcare professional able to manage medical emergencies
Elosulfase alfa is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet the initial approval criteria
Absence of unacceptable toxicity from the agent, including anaphylaxis/hypersensitivity reactions, acute respiratory complications, spinal/cervical cord compression, etc.
Individual has shown a response to therapy as evidenced by ANY ONE of the following markers when compared to pretreatment baseline values:
Stability or improvement on endurance tests
Stability or improvement in pulmonary function tests
|INDICATION(S)||DOSAGE & ADMINISTRATION|
|Mucopolysaccharidosis IVA||2 mg per kg body weight administered once every week as an intravenous infusion over a minimum of 3.5 to 4.5 hours, based on infusion volume|
LENGTH OF AUTHORIZATION
Coverage will be 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 ember'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).
Hendriksz CJ, Berger KI, Giugliani R Harmatz P, Kampmann C, Mackenzie WG,Raiman J, Villarreal MS, Savarirayan R.2015. International guidelines for the management and treatment of Morquio A syndrome.Am J Med Genet Part A 167A:11–25.
Lexi-Comp Online. (2018, February). AHFS DI. Elosulfase alfa. Retrieved February 13, 2019 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2016, August). Elosulfase alfa. Retrieved February 13, 2019 from MICROMEDEX Healthcare Series.
Regier DS, Oetgen M, Tanpaiboon P. Mucopolysaccharidosis Type IVA. 2013 Jul 11 [Updated 2016 Mar 24]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019. Available from: https://www.ncbi.nlm.nih.gov/books/NBK148668/
U. S. Food and Drug Administration. (2014, February). Center for Drug Evaluation and Research. Vimizim® (elosulfase alfa) injection, for intravenous use. Retrieved February 13, 2019 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125460s000lbl.pdf.
ORIGINAL EFFECTIVE DATE: 3/5/2014
MOST RECENT REVIEW DATE: 6/30/2019
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