Sunflower Health Plan VIMIZIM, Elosulfase Alfa Form

Elosulfase alfa (Vimizim®) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme. FDA Approved Indication Vimizim is indicated for patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). 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 Vimizim is medically necessary when the following criteria are met: I. Initial Approval Criteria A. Mucopolysaccharidosis IVA: Morquio A Syndrome (must meet all): 1. Diagnosis of Morquio A syndrome (MPS IVA) confirmed by one of the following: a. Enzyme assay demonstrating a deficiency of N-acetylgalactosamine-6-sulfatase activity; b. DNA testing; 2. Age ≥ 5 years; 3. Documentation of member’s current weight (in kg); 4. Dose does not exceed 2 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): 1. 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: Page 1 of 6 CLINICAL POLICY Elosulfase alfa CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or 2. 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 IVA: Morquio A Syndrome (must meet all): 1. 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); 2. Member is responding positively to therapy as evidenced by improvement in the individual member’s MPS IVA disease manifestation profile (see Appendix D for examples); 3. Documentation of member’s current weight (in kg); 4. If request is for a dose increase, new dose does not exceed 2 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): 1. 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 2. 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. Page 2 of 6 CLINICAL POLICY Elosulfase alfa III. Diagnoses/Indications for which coverage is NOT authorized: 1. 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 IVA: mucopolysaccharidosis IVA Appendix B: Therapeutic Alternatives Not applicable Appendix C: Contraindications/Boxed Warnings • Contraindication(s): none reported. • Boxed warning(s): risk of life-threatening anaphylactic reactions during Vimizim infusions. Appendix D: General Information The presenting symptoms and clinical course of MPS IVA can vary from one individual to another. Some examples, however, of improvement in MPS IVA disease as a result of Vimizim therapy may include improvement in: • 6-minute walking test distance • Breathing difficulties • Muscle weakness • Vision or hearing problems • Height and weight • Hepatomegaly or splenomegaly V. Dosage and Administration Indication Dosing Regimen MPS IVA 2 mg/kg IV once weekly VI. Product Availability Single-use vial: 5 mg/5 mL Maximum Dose 2 mg/kg/week VII.