ELAPRASE, Idursulfase Form

Idursulfase (Elaprase®) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme. FDA Approved Indication(s) Elaprase is indicated for the treatment of patients with Hunter syndrome (mucopolysaccharidosis [MPS] II). Elaprase has been shown to improve walking capacity in patients 5 years and older. In patients 16 months to 5 years of age, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment with Elaprase has reduced spleen volume similarly to that of adults and children 5 years of age and older. The safety and efficacy of Elaprase have not been established in pediatric patients less than 16 months of age. 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 Elaprase is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Mucopolysaccharidosis II: Hunter Syndrome (must meet all):

1. Diagnosis of MPS II (Hunter syndrome) confirmed by one of the following (a or b): a. Enzyme assay demonstrating a deficiency of iduronate 2-sulfatase activity; b. DNA testing;
   2. Age ≥ 16 months;
   3. Documentation of member’s current weight (in kg);
   4. Dose does not exceed 0.5 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): Page 1 of 6

   CLINICAL POLICY
   Idursulfase 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

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 II: Hunter 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 II (Hunter 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 0.5 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 Page 2 of 6

   CLINICAL POLICY
   Idursulfase 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 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 FVC: forced vital capacity
   Appendix B: Therapeutic Alternatives Not applicable MPS II: mucopolysaccharidosis II 6MWT: 6-minute walk test Appendix C: Contraindications/Boxed Warnings • Contraindication(s): none reported. • Boxed warning(s): risk of life-threatening anaphylactic reactions with Elaprase infusions. Appendix D: General Information • A 10% relative improvement over baseline in the percent predicted forced vital capacity (FVC) is considered by the American Thoracic Society to be a clinically significant change and not due to week-to-week variability.
   In the clinical trials of Elaprase in patients ≥ 5 years of age, patients treated with Elaprase demonstrated a 35 meter mean increase relative to placebo in the 6-minute walk test (6MWT) after 53 weeks. • • The presenting symptoms and clinical course of MPS II can vary from one individual to another. Some examples, however, of improvement in MPS II disease as a result of Elaprase therapy may include improvement in:
   • Percent predicted FVC • 6-minute walk test • Splenomegaly • Diarrhea • Joint stiffness • Growth deficiencies V. Dosage and Administration
   Indication MPS II Dosing Regimen 0.5 mg/kg IV every week Maximum Dose Based on weight Page 3 of 6

   CLINICAL POLICY
   Idursulfase VI. Product Availability
   Single-use vial: 6 mg/3 mL VII.