Pharmacy Prior Authorization Criteria

Kalydeco (ivacaftor)

Indications for Prior Authorization

Kalydeco (ivacaftor)

• For diagnosis of Cystic fibrosis
  Indicated for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

  If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Criteria

Kalydeco

*Please consult Background section for table of CFTR gene mutations responsive to Kalydeco. 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 has at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data* as detected by an FDA-cleared cystic fibrosis mutation test or a test performed at a Clinical Laboratory Improvement Amendments (CLIA)-approved facility
AND
• Patient is 1 month of age or older
AND
• Prescribed by or in consultation with one of the following:
• Specialist affiliated with a CF care center
• Pulmonologist

P & T Revisions

2025-05-01, 2025-04-30, 2025-03-25, 2025-02-04, 2024-02-01, 2023-11-23, 2023-10-03, 2023-06-27, 2023-01-06, 2022-02-03, 2021-09-27, 2021-05-19, 2021-02-04, 2020-11-03, 2020-04-01

References

1. Kalydeco Prescribing Information. Vertex Pharmaceuticals Incorporated. Boston, MA. June 2024.
2. Ramsey BW, Davies J, McElvaney G, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663-1672.

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End Notes

1. The primary efficacy endpoint in both Kalydeco pivotal trials was improvement in lung function as determined by the mean absolute change from baseline in percent predicted pre-dose FEV1 through 24 weeks of treatment. [2]

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Revision History

• 2025-05-01: Approval length updated to "Approved- no reauthorization required"
• 2025-04-30: Removing reauthorization requirement as part of extended reauthorization program.
• 2025-03-25: Removing reauthorization requirement as part of extended reauthorization program.
• 2025-02-04: Annual review: No criteria changes. Updated references.
• 2024-02-01: No criteria changes. Updated background and references.
• 2023-11-23: Addition of Kalydeco 5.8mg granules as target.
• 2023-10-03: Program update to standard reauthorization language. No changes to clinical intent
• 2023-06-27: Addition of Kalydeco 13.6mg granules and update age criteria to 1 month of age or older based on new indication
• 2023-01-06: Annual review: No criteria changes.
• 2022-02-03: Annual review: no criteria changes
• 2021-09-27: Addition of EHB formulary to guideline, no changes to criteria
• 2021-05-19: Addition of EHB formulary to guideline, no changes to criteria
• 2021-02-04: 2021 Annual Review. Added background table and edited criteria to align with updated labeling including additional CFTR mutations.
• 2020-11-03: Updated criteria due to expanded age range of 4 months and older.
• 2020-04-01: 2020 Annual review; no changes to criteria

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