FABRAZYME, Agalsidase beta Form

Agalsidase beta (Fabrazyme®) is a recombinant human alpha-galactosidase A enzyme. FDA Approved Indication(s) Fabrazyme is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. 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 Fabrazyme is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Fabry Disease (must meet all):

1. Diagnosis of Fabry disease confirmed by one of the following (a or b): a. Enzyme assay demonstrating a deficiency of alpha-galactosidase activity;
   b. DNA testing;
2. Prescribed by or in consultation with a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist;
3. Age ≥ 2 years;
   
   4. Fabrazyme is not prescribed concurrently with Galafold®;
   5. Dose does not exceed 1 mg/kg every 2 weeks. 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):

4. 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
   Agalsidase Beta 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

5. 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. Fabry Disease (must meet all):
6. 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);
7. Member is responding positively to therapy as evidenced by improvement in the individual member’s Fabry disease manifestation profile (see Appendix D for examples);
8. If request is for a dose increase, new dose does not exceed 1 mg/kg every 2 weeks. 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):
9. 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

10. 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
    Agalsidase Beta 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.
    IV. Appendices/General Information Appendix A: Abbreviation/Acronym Key FDA: Food and Drug Administration Appendix B: Therapeutic Alternatives Not applicable Appendix C: Contraindications/Boxed Warnings None reported Appendix D: General Information The presenting symptoms and clinical course of Fabry disease can vary from one individual to another. As such, there is not one generally applicable set of clinical criteria that can be used to determine appropriateness of continuation of therapy. Some examples, however, of improvement in Fabry disease as a result of Fabrazyme therapy may include improvement in:
    • Fabry disease signs such as pain in the extremities, hypohidrosis or anhidrosis, or angiokeratomas • Diarrhea, abdominal pain, nausea, vomiting, and flank pain • Renal function • Neuropathic pain, heat and cold intolerance, vertigo and diplopia • Fatigue • Cornea verticillata V. Dosage and Administration
    Indication Fabry disease Dosing Regimen 1 mg/kg IV every 2 weeks
    Maximum Dose 1 mg/kg/2 weeks VI. Product Availability
    Single-use vials: 5 mg, 35 mg VII.