69794-0001-XX Mepsevii 10 mg SOL (Ultragenyx Pharmaceutical Inc.)
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.
Vestronidase alfa-vjbk is considered medically appropriate if ALL of the following criteria are met:
Pediatric (5 months of age or older) or adult Individual
Diagnosis of Mucopolysaccharidos is VII (MPS VII, Sly syndrome) confirmed by BOTH of the following:
Beta-glucuronidase enzyme deficiency in peripheral blood leukocytes
Detection of pathogenic mutations in the GUSB gene by molecular genetic testing
Documentation of ANY ONE of the following:
Baseline 6-minute walk test (6-MWT)
Baseline balance and gross motor proficiency as assessed by the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
Baseline urinary excretion of glycosaminoglycans (GAGs), including chondroitin sulfate and dermatan sulfate
Vestronidase alfa-vjbk is considered medically appropriate for renewal if ALL of the following criteria are met:
Disease response to treatment as defined by improvement in ANY ONE of the following:
6-minute walk test (6-MWT)
Balance and gross motor proficiency as assessed by the Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
Urinary excretion of GAGs, including chondroitin sulfate and dermatan sulfate
Absence of unacceptable toxicity from the agent, including, but not limited to, anaphylaxis and allergic reactions; immune mediated reactions; acute respiratory complications; acute cardiorespiratory reactions; infusion reactions
DOSAGE & ADMINISTRATION
Mucopolysaccharidosis VII (MPS VII, Sly syndrome)
4 mg/kg every two weeks as an intravenous infusion
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed.
Click here to view DOSAGE LIMITS
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).
National Organization for Rare Disorders (NORD). (2017). Mucopolysaccharidosis type vii, diagnosis. Retrieved December 12, 2017 from https://rarediseases.org/rare-diseases/sly-syndrome/.
U. S. Food and Drug Administration. (2017, November). Center for Drug Evaluation and Research. Mepsevii™ (vestronidase alfa-vjbk) injection, for intravenous use. Retrieved December 6, 2017 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761047s000lbl.pdf.
ORIGINAL EFFECTIVE DATE: 1/18/2018
MOST RECENT REVIEW DATE: 1/18/2018
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