ELELYSO, Taliglucerase Alfa Form

Taliglucerase alfa (Elelyso®) is a hydrolytic lysosomal glucocerebroside-specific enzyme.
FDA Approved Indication(s) Elelyso is indicated for the treatment of patients 4 years and older with a confirmed diagnosis of type 1 Gaucher 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 Elelyso 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 ≥ 4 years;
   3. Member is symptomatic (e.g., anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly);
   4. One of the following (a or b):
      a. For GD1: Failure of Cerezyme® and Cerdelga® unless clinically significant adverse effects are experienced or both are contraindicated;
      b. For GD3: Failure of Cerezyme, unless contraindicated or clinically significant adverse effects are experienced;
      *Prior authorization may be required for Cerezyme and Cerdelga
3. Elelyso is not prescribed concurrently with VPRIV® (velaglucerase alfa) or Cerezyme® (imiglucerase);

4. Documentation of member’s current weight (in kg);

   7. Dose does not exceed 60 units/kg every two weeks. Approval duration:
      HIM/Medicaid – 6 months Commercial – 6 months or to the member’s renewal date, whichever is longer
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   CLINICAL POLICY
   Taliglucerase Alfa 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): 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. Elelyso is not prescribed concurrently with VPRIV (velaglucerase alfa) or Cerezyme (imiglucerase);
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 2 weeks. Approval duration:
       HIM/Medicaid – 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: Page 2 of 7

    CLINICAL POLICY
    Taliglucerase Alfa 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

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 GD1: type 1 Gaucher disease
    GD3: type 3 Gaucher disease Appendix B: Therapeutic Alternatives This table provides a listing of preferred alternative therapy recommended in the approval criteria. The drugs listed here may not be a formulary agent for all relevant lines of business and may require prior authorization.
    Dosing Regimen Drug Name Dose Limit/Maximum Dose CYP2D6 EM, IM: 168 mg/day CYP2D6 PM: 84 mg/day 60 U/kg every 2 weeks Cerdelga (eliglustat) Cerezyme (imiglucerase) GD1: CYP2D6 EM, IM: 84 mg PO BID CYP2D6 PM: 84 mg PO QD GD1 or GD3:
    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 Therapeutic alternatives are listed as Brand name and generic (Brand name ® ) when the drug is available by both brand and generic
    ® (generic) when the drug is available by brand name only Appendix C: Contraindications/Boxed Warnings None reported Page 3 of 7

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    Taliglucerase Alfa 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 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 Dosing Regimen Gaucher disease Treatment-naïve patients: 60 units/kg IV every other week
    Patients switching from imiglucerase:
    Begin at the same unit/kg dose as the patient’s previous imiglucerase dose; administer every other week
    VI. Product Availability
    Single-use vial: 200 units Maximum Dose Individualized (adjust based on achievement and maintenance of therapeutic goals) VII.