Subject: Orkambi 1885-A SGM P2022

Drug

ORKAMBI  (lumacaftor/livacaftor)

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 age 2 years 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: genetic testing report confirming the presence of the appropriate CFTR gene mutation. 

III. 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 gene.
B.   The member is positive for the F508del mutation on both alleles of the CFTR gene.
C.   The member is at least 2 years of age.
D.   Orkambi will not be used in combination with other medications containing ivacaftor. 

IV. CONTINUATION OF THERAPY 

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in Section III 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.; July 2019.

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 26, 2022