Pharmacy Prior Authorization Criteria

Orkambi (lumacaftor/ivacaftor)

Indications for Prior Authorization

Orkambi (lumacaftor/ivacaftor)

• For diagnosis of Cystic fibrosis (CF)
  Indicated for the treatment of cystic fibrosis (CF) in patients age 1 years and older who are homozygous for the F508del mutation in the 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.

Criteria

Orkambi (100 mg - 125 mg) tablet

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of cystic fibrosis (CF)
AND
• Patient is homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene as detected by an FDA-cleared cystic fibrosis mutation test or Clinical Laboratory Improvement Amendments (CLIA)-approved facility
AND
• Patient is 6 years of age or older
AND
• Prescribed by or in consultation with one of the following:
• Specialist affiliated with a cystic fibrosis care center
• Pulmonologist

Orkambi (200 mg - 125 mg) tablet

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of cystic fibrosis (CF)
AND
• Patient is homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene as detected by an FDA-cleared cystic fibrosis mutation test or Clinical Laboratory Improvement Amendments (CLIA)-approved facility
AND
• Patient is 12 years of age or older
AND
• Prescribed by or in consultation with one of the following:
• Specialist affiliated with a cystic fibrosis care center
• Pulmonologist

Orkambi (100 mg - 125 mg) tablet, Orkambi (200 mg - 125 mg) tablet

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient demonstrates positive clinical response to therapy (i.e., improvement in lung function [forced expiratory volume in one second {FEV1}], decreased number of pulmonary exacerbations)

Orkambi (100 mg - 125 mg) granules packet, Orkambi (150 mg - 188 mg) granules packet, Orkambi (75 mg - 94 mg) granules packet

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of cystic fibrosis (CF)
AND
• Patient is homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene as detected by an FDA-cleared cystic fibrosis mutation test or Clinical Laboratory Improvement Amendments (CLIA)-approved facility
AND
• One of the following:
• Patient is 1 through 5 years of age
OR
• Both of the following:
• Patient is 6 years of age or greater
• Patient is unable to swallow oral tablets
AND
• Prescribed by or in consultation with one of the following:
• Specialist affiliated with a cystic fibrosis care center
• Pulmonologist

Orkambi (100 mg - 125 mg) granules packet, Orkambi (150 mg - 188 mg) granules packet, Orkambi (75 mg - 94 mg) granules packet

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient demonstrates positive clinical response to therapy (i.e., improvement in lung function [forced expiratory volume in one second {FEV1}], decreased number of pulmonary exacerbations)
AND
• One of the following:
• Patient is 1 through 5 years of age
OR
• Both of the following:
• Patient is 6 years of age or greater
• Patient is unable to swallow oral tablets

P & T Revisions

2024-03-22, 2023-12-20, 2023-10-10, 2023-04-06, 2022-10-04, 2022-04-04, 2021-09-27, 2021-05-21, 2021-04-07, 2020-04-01

References

1. Orkambi Prescribing Information. Vertex Pharmaceuticals Incorporated. Boston, MA. August 2023.

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End Notes

1. The primary efficacy endpoint in both pivotal trials was improvement in lung function as determined by the mean absolute change from baseline in percent predicted FEV1 through 24 weeks of treatment. [1]

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Revision History

• 2024-03-22: Annual review: No criteria changes. Updated references.
• 2023-12-20: Patient demonstrates positive clinical response to therapy.
• 2023-10-10: Patient demonstrates positive clinical response to therapy.
• 2023-04-06: Annual review: No criteria changes. Updated references.
• 2022-10-04: Added Orkambi 75mg-94mg granule packet to guideline. Changed age criterion where appropriate to align with PI. Updated background and references.
• 2022-04-04: Annual review: no criteria changes.
• 2021-09-27: Addition of EHB formulary to guideline, no changes to criteria
• 2021-05-21: Addition of EHB formulary to guideline, no changes to criteria
• 2021-04-07: 2021 Annual Review. No changes to criteria.
• 2020-04-01: 2020 Annual review; no changes to criteria

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