Subject: Orkambi 1885-A SGM P2023

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 a combination of lumacaftor and ivacaftor 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 no
          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 Inc.; September 2022.

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 11, 2023