MEPSEVII, Vestronidase alfa-vjbk Form

Vestronidase alfa-vjbk (Mepsevii®) is a recombinant human lysosomal beta glucuronidase enzyme replacement therapy. FDA Approved Indication(s) Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). Limitation(s) of use: The effect of Mepsevii on the central nervous system manifestations of MPS VII has not been determined. Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria.
It is the policy of health plans affiliated with Centene Corporation® that Mepsevii is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Mucopolysaccharidosis VII: Sly Syndrome (must meet all):

1. Diagnosis of MPS VII (Sly syndrome) confirmed by one of the following (a or b): a. Two repeated enzyme assay tests demonstrating a deficiency of beta- glucuronidase; b. One DNA testing showing GUSB gene mutation;
2. Prescribed by or in consultation with a specialist with expertise in lysosomal storage diseases (e.g., pediatric endocrinologist, pediatric geneticist);
3. Apparent clinical signs of lysosomal storage disease including at least one of the following (a, b, c, or d): a. Enlarged liver and spleen; b. Joint limitations; c. Airway obstruction or pulmonary problems; d. Limitations of mobility;

4. Dose does not exceed 4 mg/kg IV every 2 weeks. Approval duration: 6 months Page 1 of 6

   CLINICAL POLICY Vestronidase Alfa-vjbk B. Other diagnoses/indications (must meet 1 or 2):

5. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or
6. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   II. Continued Therapy A. Mucopolysaccharidosis VII: Sly Syndrome (must meet all):
7. Member meets one of the following (a or b): a. Currently receiving medication via Centene benefit or member has previously met initial approval criteria; b. Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B);
8. Member is responding positively to therapy as evidenced by improvement in the individual member’s MPS VII disease manifestation profile (see Appendix D for examples);
9. If request is for a dose increase, new dose does not exceed 4 mg/kg IV every 2 weeks.
   Approval duration: 12 months B. Other diagnoses/indications (must meet 1 or 2):
10. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

11. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND Page 2 of 6

    CLINICAL POLICY Vestronidase Alfa-vjbk criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
    III. Diagnoses/Indications for which coverage is NOT authorized:
    A. Non-FDA approved indications, which are not addressed in this policy, unless there is sufficient documentation of efficacy and safety according to the off label use policies – CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid, or evidence of coverage documents. IV. Appendices/General Information Appendix A: Abbreviation/Acronym Key FDA: Food and Drug Administration MPS VII: Mucopolysaccharidosis VII Appendix B: Therapeutic Alternatives
    Not applicable Appendix C: Contraindications/Boxed Warnings • Contraindication(s): none reported • Boxed warning(s): anaphylaxis Appendix D: General Information
    • The presenting symptoms and clinical course of MPS VII can vary from one individual to another. Some examples, however, of improvement in MPS VII disease as a result of Mepsevii therapy may include improvement in: o 6-minute walking distance o Breathing difficulties o Muscle weakness o Vision or hearing problems o Hepatomegaly or splenomegaly o Reduction of total urinary glycosaminoglycan (uGAG) excretion o Stair climbing capacity as measured by the 3 Minute Stair Climb Test o Height and weight growth velocity compared to estimated pretreatment growth rate velocity from medical records for pediatric patients • In individuals with MPS, the circulation of fluid through the blood-brain barrier may become blocked, which can lead to hydrocephalus and cortical atrophy. Seizures are a complication most common among individuals with severe forms of MPS. The clinical benefit on this central nervous system manifestation with treatment of Mepsevii has not yet been determined.
    V. Dosage and Administration
    Indication MPS VII (Sly syndrome) Dosing Regimen 4 mg/kg IV every 2 weeks Maximum Dose 4 mg/kg/2 weeks Page 3 of 6

    CLINICAL POLICY Vestronidase Alfa-vjbk VI. Product Availability
    Single-dose vial: 10 mg/5 mL VII.