Subject: Orkambi 1885-A SGM P2024

Drug

ORKAMBI  (lumacaftor/ivacaftor)

Policy:

      I.  INDICATIONS

          The indications below including FDA-approved indications and compendial uses are
          considered a covered benefit provided that all the approval criteria are met and the member
          has no exclusions to the prescribed therapy.

          FDA-Approved Indication
          Orkambi is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older
          who are homozygous for the F508del mutation in the cystic fibrosis transmembrane
          conductance regulator (CFTR) gene. If the patient’s genotype is unknown, an FDA-cleared CF
          mutation test should be used to detect the presence of the F508del mutation on both alleles of
          the CFTR gene.

          Limitation of use: The efficacy and safety of Orkambi have not been established in patients
          with CF other than those homozygous for the F508del mutation.

          All other indications are considered experimental/investigational and are not medically
          necessary.


    II.  DOCUMENTATION

          Submission of the following information is necessary to initiate the prior authorization review:
          For initial requests, genetic testing report confirming the presence of the appropriate CFTR
          gene mutation.


   III.  PRESCRIBER SPECIALTIES

          This medication must be prescribed by or in consultation with a pulmonologist.


   IV.  CRITERIA FOR INITIAL APPROVAL

          Cystic fibrosis
          Authorization of 12 months may be granted for treatment of cystic fibrosis when all of the
          following criteria are met:
          A.  Genetic testing was conducted to detect a mutation in the CFTR
          B.  The member is positive for the F508del mutation on both alleles of the CFTR
          C.  The member is at least 1 year of age.
          D.  Orkambi will not be used in combination with other medications containing ivacaftor.


    V.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for continued treatment in members requesting
          reauthorization for an indication listed in Section IV who are experiencing benefit from therapy
          as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from
          baseline).

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Orkambi [package insert]. Boston, MA: Vertex Pharmaceuticals Incorporated; August 2023.

 

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

June 09, 2024