NAGLAZYME, Galsulfase Form

Galsulfase (Naglazyme®) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme. FDA approved indication Naglazyme is indicated for the treatment of patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). Naglazyme has been shown to improve walking and stair-climbing capacity. 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 Naglazyme is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Mucopolysaccharidosis VI: Maroteaux-Lamy Syndrome (must meet all):

1. Diagnosis of MPS VI (Maroteaux-Lamy syndrome) confirmed by one of the following (a or b): a. Enzyme assay demonstrating a deficiency in N-acetylgalactosamine 4-sulfatase (arylsulfatase B) activity; b. DNA testing;
   2. Age ≥ 3 months;
   3. Documentation of member’s current weight (in kg);
   4. Dose does not exceed 1 mg/kg per week. Approval duration:
      Medicaid/HIM – 6 months Commercial – 6 months or to the member’s renewal date, whichever is longer B. Other diagnoses/indications (must meet 1 or 2):

2. 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 Page 1 of 6

   CLINICAL POLICY
   Galsulfase 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

3. 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 VI: Maroteaux-Lamy Syndrome (must meet all):
4. 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);
5. Member is responding positively to therapy as evidenced by improvement in the individual member’s MPS VI (Maroteaux-Lamy syndrome) manifestation profile (see Appendix D for examples);
6. Documentation of member’s current weight (in kg);
   4. If request is for a dose increase, new dose does not exceed 1 mg/kg per week. Approval duration:
      Medicaid/HIM – 12 months Commercial – 6 months or to the member’s renewal date, whichever is longer B. Other diagnoses/indications (must meet 1 or 2):
7. 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

8. 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.
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   CLINICAL POLICY
   Galsulfase 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 policy – 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 VI: mucopolysaccharidosis VI
   Appendix B: Therapeutic Alternatives Not applicable Appendix C: Contraindications/Boxed Warnings None reported Appendix D: General Information
   The presenting symptoms and clinical course of MPS VI can vary from one individual to another. Some examples, however, of improvement in MPS VI disease as a result of Naglazyme therapy may include improvement in:
   • 12-minute walking test distance; • 3-minute stair climb rate; • Poor endurance; • Vision problems; • Respiratory infections; • Breathing problems, sleep apnea; • High blood pressure; • Joint stiffness; • Height and weight; • Hepatomegaly, splenomegaly. V. Dosage and Administration
   Indication Dosing Regimen MPS VI 1 mg/kg IV once weekly VI. Product Availability
   Vial: 5 mg/5 mL Maximum Dose 1 mg/kg/week VII.