ARIKAYCE, Amikacin Sulfate Liposome Form

Amikacin (Arikayce®) is a liposomal formulation of amikacin – an aminoglycoside antibiotic active against aerobic gram-negative rods. FDA Approved Indication(s) Arikayce is indicated in adults who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for Arikayce are currently available, reserve Arikayce for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients. This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Limitation(s) of use: Arikayce has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of Arikayce is not recommended for patients with non-refractory MAC lung 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 Arikayce is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Mycobacterium Avium Complex (must meet all):

1. Diagnosis of MAC;

   2. Prescribed by or in consultation with an infectious disease specialist or pulmonologist;
   3. Age ≥ 18 years;
   4. Failure, as evidenced by positive sputum culture, of at least a 6-month trial of a multidrug background regimen therapy at up to maximally indicated doses (see Page 1 of 6

   CLINICAL POLICY Amikacin Appendix B), unless all are contraindicated or clinically significant adverse effects are experienced;

2. Dose does not exceed one vial per day. Approval duration: 6 months B. Other diagnoses/indications (must meet 1 or 2):
3. 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
4. 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. Mycobacterium Avium Complex (must meet all):
   
   1. Currently 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. Documentation of at least 3 consecutive negative monthly sputum cultures in the first 6 months of therapy or at least 2 consecutive negative monthly sputum cultures in the last 2 months of therapy;
6. Member has not received more than 12 months of treatment following conversion to negative sputum status;
7. If request is for a dose increase, new dose does not exceed one vial per day.
   Approval duration: Up to a total of 12 months of treatment after converting to negative sputum status B. Other diagnoses/indications (must meet 1 or 2):

8. 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 6

   CLINICAL POLICY Amikacin 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

9. 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 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 MAC: mycobacterium avium complex 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.
   Drug Name Dosing Regimen Dose Limit/ Maximum Dose Combo used for initial therapy for nodular/bronchiectasis disease Combo used for initial therapy for cavitary disease clarithromycin (Biaxin®) or azithromycin (Zmax®) + ethambutol (Myambutol®) + rifampin (Rifadin®) Variable dosing Variable dosing clarithromycin (Biaxin®) or azithromycin (Zmax®) + ethambutol (Myambutol®) + rifampin (Rifadin®) + streptomycin or amikacin (Amikin®) or none. clarithromycin (Biaxin®) or azithromycin (Zmax®) + ethambutol (Myambutol®) + rifampin (Rifadin®) or rifabutin (Mycobutin®) + streptomycin or amikacin (Amikin®) Therapeutic alternatives are listed as Brand name® (generic) when the drug is available by brand name only and generic (Brand name®) when the drug is available by both brand and generic. Combo used for advanced (severe) or previously treated disease Variable dosing Page 3 of 6

   CLINICAL POLICY Amikacin Appendix C: Contraindications/Boxed Warnings • Contraindication(s): a known hypersensitivity to any aminoglycoside. • Boxed warning(s): risk of increased respiratory adverse reactions, including, hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalization in some cases. V. Dosage and Administration
   Indication Dosing Regimen MAC Inhalation of the contents of one 590 mg/8.4 mL Arikayce vial per day Maximum Dose 590 mg/8.4 mL per day VI. Product Availability
   Solution for inhalation: 590 mg/8.4 mL VII.