CEREZYME, Imiglucerase Form

Imiglucerase (Cerezyme®) is an analogue of the human enzyme ß-glucocerebrosidase. FDA Approved Indication(s) Cerezyme is indicated for treatment of adult and pediatric patients 2 years of age and older with type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, or hepatomegaly or splenomegaly. 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 Cerezyme is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Gaucher Disease (must meet all):

1. Diagnosis of type 1 (GD1) or type 3 Gaucher disease (GD3) confirmed by one of the following (a or b): a. Enzyme assay demonstrating a deficiency of beta-glucocerebrosidase (glucosidase) activity; b. DNA testing;
2. Age ≥ 2 years;
   3. Member is symptomatic (e.g., anemia, thrombocytopenia, bone disease,
3. Cerezyme is not prescribed concurrently with VPRIV® (velaglucerase alfa) or hepatomegaly, splenomegaly); Elelyso® (taliglucerase alfa);
4. Documentation of member’s current weight (in kg);
   6. Dose does not exceed 60 units/kg every two 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):

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): Page 1 of 6

   CLINICAL POLICY
   Imiglucerase 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. Gaucher Disease (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 increased or stabilized platelet count or hemoglobin, reduced or stabilized spleen or liver volume, or decreased bone pain;
9. Cerezyme is not prescribed concurrently with VPRIV (velaglucerase alfa) or Elelyso (taliglucerase alfa);
10. Documentation of member’s current weight (in kg);
    5. If request is for a dose increase, new dose does not exceed 60 units/kg every two 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):

11. 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 2 of 6

    CLINICAL POLICY
    Imiglucerase CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

12. 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.
    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 ERT: enzyme replacement therapy FDA: Food and Drug Administration Appendix B: Therapeutic Alternatives Not applicable Appendix C: Contraindications/Boxed Warnings None reported GD1: type 1 Gaucher disease GD3: type 3 Gaucher disease Appendix D: General Information • Measures of therapeutic response: GD1 is a heterogeneous disorder which involves the visceral organs, bone marrow, and bone in almost all affected patients. Common conditions resulting from GD1 include anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone disease. Therefore, hemoglobin level, platelet count, liver volume, spleen volume, and bone pain are clinical parameters that can indicate therapeutic response to GD1 therapies. In some clinical trials, stability has been defined as the following thresholds of change from baseline: hemoglobin level < 1.5 g/dL decrease, platelet count < 25% decrease, liver volume < 20% increase, and spleen volume < 25% increase. • Enzyme replacement therapy such as Cerezyme may have beneficial palliative effects in Type 2 disease, but does not alter the outcome and is not generally used. • According to the European consensus guidelines revised recommendations on the management of neuronopathic Gaucher disease by Vellodi et al: (1) there is clear evidence in most patients that enzyme replacement therapy (ERT) ameliorates systemic involvement in non-neuronopathic (type 1) as well as chronic neuronopathic Gaucher disease (type 3), enhancing quality of life; (2) There is no evidence that ERT has reversed, stabilized or slowed the progression of neurological involvement; (3) In patients with established acute neuronopathic Gaucher disease (type 2), enzyme replacement Page 3 of 6

    CLINICAL POLICY
    Imiglucerase therapy has had little effect on the progressively downhill course. It has merely resulted in prolongation of pain and suffering. • There is currently insufficient clinical evidence that supports the combination use of enzyme replacement therapy with Zavesca® (miglustat) or Cerdelga® (eliglustat), or concurrent use of two or more enzyme replacement therapies at once.
    V. Dosage and Administration
    Indication Gaucher disease Dosing Regimen Recommended dosage based upon disease severity ranges from 2.5 U/kg via IV infusion 3 times a week to 60 U/kg once every 2 weeks; titrate the dosage based on clinical manifestations of disease and therapeutic goals for the patient
    Maximum Dose 60 U/kg every 2 weeks VI. Product Availability
    Vial: 400 units VII.