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    Mepsevii™

    Drug - Mepsevii™ (vestronidase alfa injection) [Ultragenyx Pharmaceutical Inc.]

    July 2018

    Therapeutic area - Metabolic Disease, Enzyme Replacement

    Initial approval criteria

    • Patient must be aged 5 months or older AND
    • Patient must be prescribed Mepsevii by, or in consultation with, a metabolic or genetic specialist AND
    • Patient must have a documented diagnosis of mucopolysaccharidosis VII (MPS VII, also known as Sly Syndrome) 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
    • Patient must have documented baseline value for at least ONE of the following:
      • 6 minute walk test (6MWT) and/or motor function [e.g., Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)] OR
      • Liver and/or spleen volume OR
      • Urinary excretion of glycosaminoglycans (GAGs) such as chondroitin sulfate and dermatan sulfate OR
      • Skeletal involvement OR
      • Pulmonary function tests

    Renewal criteria

    • Patient has absence of unacceptable toxicity from the drug, including any of the following: anaphylaxis and severe allergic reactions, etc. AND
    • Patient must have demonstrated response to therapy compared to pretreatment baseline in at least ONE of the following:
      • Stability or improvement in 6MWT and/or motor function OR
      • Reduction in liver and/or spleen volume OR
      • Reduction in urinary excretion of glycosaminoglycans (GAGs) OR
      • Stability of skeletal disease OR
      • Stability or improvement in pulmonary function tests

    Quantity limits

    • 46 vials per 14 days

    Background

    The effect of Mepsevii on the central nervous system manifestations of MPS VII has not been determined.

    Billing of Mepsevii

    Mepsevii is not covered through the MHCP fee-for-service pharmacy benefit and must be submitted as a medical claim. Mepsevii should be administered by a healthcare professional in a healthcare setting. In line with clinical practice, monitoring of patients after administration of Mepsevii is recommended by the manufacturer.

    Questions?

    MHCP Provider Call Center 651-431-2700 or 800-366-5411

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