Subject: Orkambi SGM 1885-A P2025

Drug

ORKAMBI  (lumacaftor/ivacaftor)

Policy:

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 Indication¹

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.

Limitations 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.

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.

Prescriber Specialties

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

Coverage criteria

Cystic Fibrosis¹

Authorization of 12 months may be granted for treatment of cystic fibrosis when all of the following criteria are met:

• Genetic testing was conducted to detect a mutation in the CFTR gene.
• The member is homozygous for the F508del mutation (positive for the F508del mutation on both alleles) of the CFTR gene.
• The member is at least 1 year of age.

Continuation of Therapy

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

Other

Orkambi will not be used in combination with another CFTR modulator for the treatment of cystic fibrosis (e.g., Alyftrek, Symdeko).

Place of Service:

Outpatient

The above policy is based on the following references:

1. Orkambi [package insert]. Boston, MA: Vertex Pharmaceuticals Incorporated; December 2024.

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 06, 2025