69794-0001-XX MEPSEVII 2MG/ML Solution (ULTRAGENYX PHARMACEUTICAL)
Vestronidase alfa-vjbk is a recombinant human lysosomal beta glucuronidase which is a purified human enzyme produced by recombinant DNA technology. Its amino acid sequence is the same as that of naturally occurring human beta-glucuronidase (GUS).
Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS which results in the accumulation of glycosaminoglycans (GAGs) throughout the body leading to multisystem tissue and organ damage. Enzyme replacement therapy with vestronidase alfa-vjbk has been shown to reduce urinary excretion of GAGs including chondroitin sulfate and dermatan sulfate, which was sustained with continued treatment.
Vestronidase alfa-vjbk is considered medically necessary for the treatment of mucopolysaccharidosis VII (MPS VII or Sly syndrome) if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Vestronidase alfa-vjbk for the treatment of other conditions/diseases is considered investigational.
Patient is at least 5 months of age; AND
Mucopolysaccharidosis VII (MPS VII; Sly syndrome)
Patient has a definitive diagnosis of MPS VII confirmed by BOTH of the following:
Beta-glucuronidase enzyme deficiency in peripheral blood leukocytes; AND
Detection of pathogenic mutations in the GUSB gene by molecular genetic testing; AND
Documented baseline value for one or more of the following: six minute walk test (6MWT), motor function [i.e., Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)], liver and/or spleen volume, urinary excretion of glycosaminoglycans (GAGs) such as chondroitin sulfate and dermatan sulfate, skeletal involvement (i.e. Z-score), pulmonary function tests, shoulder flexion, visual acuity, etc
Patient continues to meet indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in the Initial Approval Criteria; AND
Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: anaphylaxis and severe allergic reactions, etc.; AND
Patient has responded to therapy compared to pretreatment baseline in one or more of the following:
Stability or improvement in 6MWT, shoulder flexion, visual acuity, and/or other motor functions
Reduction in liver and/or spleen volume
Reduction in urinary excretion of GAGs
Stability of skeletal disease (i.e. improvement in Z-score)
Stability or improvement in pulmonary function tests
Mucopolysaccharidosis VII (Sly syndrome)
4 mg/kg administered as an intravenous (IV) infusion once every 2 weeks
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed.
Max Units (per dose and over time) [HCPCS Unit]:
460 billable units (460 mg) every 14 days
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).
1. Mepsevii [package insert]. Novato, CA; Ultragenyx Pharmaceutical Inc.; December 2020. Accessed December 2020.
2. Montaño AM, Lock-Hock N, Steiner RD, et al. Clinical course of sly syndrome (mucopolysaccharidosis type VII). J Med Genet. 2016 Jun;53(6):403-18.
3. Harmatz P, Whitley CB, Wang RY, et al. A novel, randomized, placebo-controlled, blindstart, single-crossover phase 3 study to assess the efficacy and safety of UX003 (rhGUS) enzyme replacement therapy in patients with MPS VII. Mol Genet Metab. 2017;120:S63.
4. Qi Y, McKeever K, Taylor J, et al. Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials. Clin Pharmacokinet. 2019 May;58(5):673-683. doi: 10.1007/s40262-018-0721-y.
5. Lexicomp Online. (2021). AHFS DI. Vestronidase alfa-vjbk. Retrieved February 10, 2021 from Lexicomp Online with AHFS.
6. MICROMEDEX Healthcare Series. Drugdex Evaluations. (2019, December). Vestronidase alfa-vjbk. Retrieved February 10, 2021 from MICROMEDEX Healthcare Series.
ORIGINAL EFFECTIVE DATE: 1/18/2018
MOST RECENT REVIEW DATE: 3/9/2021
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