WHA

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

For initial authorization request, approve through 12/31/2039 For reauthorization request, bypass criteria review and approve through 12/31/2039

Prior Authorization

Length of Approval: When approved; no reauthorization required

  • 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

For initial authorization request, approve through 12/31/2039 For reauthorization request, bypass criteria review and approve through 12/31/2039

Prior Authorization

Length of Approval: When approved; no reauthorization required

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

For initial authorization request, approve through 12/31/2039 For reauthorization request, bypass criteria review and approve through 12/31/2039

Prior Authorization

Length of Approval: When approved; no reauthorization required

  • 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

  • Guideline ID
    GL-249284

  • Formulary
    premium

  • Effective date
    May 1, 2025

  • P&T approval date

P & T Revisions

2025-05-01, 2025-04-30, 2025-03-25, 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

  1. Orkambi Prescribing Information. Vertex Pharmaceuticals Incorporated. Boston, MA. August 2023.
  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]
  • 2025-05-01: Approval length updated to "Approved- no reauthorization required"
  • 2025-04-30: Note and approval lenght updated from 2099 to 2039
  • 2025-03-25: Removing reauthorization requirement as part of extended reauthorization program.
  • 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