CurrentUnitedHealthcarePolicy N/A

Kalydeco (ivacaftor) prior authorization

Prior authorization and medical necessity criteria for coverage of Kalydeco (ivacaftor) for treatment of cystic fibrosis in eligible members; applies to UnitedHealthcare pharmacy medical benefit processing.

Policy Summary

PayerUnitedHealthcare

PolicyKalydeco (ivacaftor) prior authorization

Policy CodePolicy N/A

Change TypeNo material change

Effective DateSep 1, 2025

Next Review DateN/A

Key ActionSubmit laboratory results confirming at least one CFTR mutation responsive to ivacaftor when requesting prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsAuthorization duration

≥1 monthFDA-indicated age

Prior AuthProgram type

Annual P&TPolicy review history

Coverage Criteria for Kalydeco (ivacaftor)

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Initial Authorization: Diagnosis of cystic fibrosis (CF).

Initial Authorization: Submission of laboratory results confirming the patient has at least one CFTR gene mutation that is responsive to Kalydeco (see Kalydeco Prescribing Information for a complete up‑to‑date mutation list).

Complex/compound mutations noted; reference prescribing information for full list and clinical data.

Initial Authorization: Prescribed by or in consultation with a provider who specializes in the treatment of cystic fibrosis.

inv-02: Reauthorization

Covered when ALL of the following are met:

Reauthorization: Documentation of positive clinical response to Kalydeco therapy (e.g., improved or stable lung function).

Authorization will be issued for 12 months.

Patients who do not have a CFTR gene variant that is responsive to ivacaftor (as identified in the Kalydeco prescribing information or by clinical/in‑vitro assay data) are excluded from coverage under these criteria. Documentation confirming at least one responsive mutation is required for approval; absence of such documentation may result in denial of the request.

Initial Therapy Requirements

inv-11: Initial Therapy

Initial therapy requirements

Initial therapy: Diagnosis of cystic fibrosis and laboratory confirmation of at least one CFTR mutation responsive to ivacaftor; if genotype is unknown, use an FDA‑cleared CF mutation test with bi‑directional sequencing when recommended by the test instructions.

See Kalydeco prescribing information for the full responsive mutation table and details on complex/compound mutations.

Continuation / Reauthorization Criteria

inv-12: Continuation Therapy

Reauthorization/Continuation rules

Continuation: Require documentation of positive clinical response to therapy (e.g., improved or stable lung function).

Authorizations are issued for 12 months; previous prescriber restriction for reauthorization removed—current requirement is clinical response only.

Provider Actions and Documentation Requirements

Prior Authorization

Initial authorization required

Initial authorization is required. Member must have a diagnosis of cystic fibrosis and submission of laboratory results confirming at least one CFTR mutation that is responsive to Kalydeco. If the patient's genotype is unknown, an FDA‑cleared CF mutation test should be used and followed by verification with bi‑directional sequencing when recommended by the test instructions for use. A complete, up‑to‑date list of responsive mutations can be referenced in the Kalydeco Prescribing Information.

Diagnosis: Cystic fibrosis (CF).
Required lab confirmation: Documentation of at least one CFTR mutation responsive to ivacaftor per Kalydeco prescribing information.
Genotype unknown guidance: Perform an FDA‑cleared CF mutation test and verify with bi‑directional sequencing when recommended.

Step Therapy

Step therapy

No step therapy requirements are specified for Kalydeco in this policy. Prescribers should still check benefit plan-specific rules and any supply limits that may apply.

Documentation Required

Required documentation

Submit laboratory results confirming at least one responsive CFTR mutation with the initial prior authorization request. If genotype is unknown at time of request, include documentation that an FDA‑cleared CF mutation test was performed and any follow-up sequencing results when available.

Include copy of genetic test report showing the responsive CFTR mutation(s).
If initial testing is pending, document testing ordered and expected date of results.

Documentation Required

Reauthorization documentation

For reauthorization, provide documentation of a positive clinical response to Kalydeco therapy, such as improved or stable lung function or other clinical benefit documented in the medical record.

Examples: spirometry results showing improved or stable FEV1, clinical notes describing symptomatic improvement, weight gain, reduced pulmonary exacerbations, or other objective measures of benefit.

Denial Risk

Denial triggers

Denial may occur if there is no diagnosis of cystic fibrosis or if laboratory confirmation of a CFTR mutation responsive to Kalydeco is not provided. Requests that lack required genetic documentation or documentation of clinical benefit for continuation may be denied.

Denial triggers: absence of CF diagnosis; no lab report confirming a responsive CFTR mutation; genotype unknown with no documentation of testing ordered or results.
For reauthorization: lack of documented clinical response to therapy may result in denial.

Definitions

Responsive CFTR mutation — definition and source

DefinitionA CFTR gene variant listed in the Kalydeco (ivacaftor) prescribing information or otherwise identified by clinical and/or in vitro assay data as responsive to ivacaftor therapy.

Source — prescribing informationComplete, up‑to‑date list of responsive CFTR mutations is referenced to the Kalydeco Prescribing Information.

Source — clinical/in‑vitro dataResponsiveness may be established by clinical trial evidence or in vitro assay data demonstrating benefit with ivacaftor.

Unknown genotype pathwayIf genotype is unknown, an FDA‑cleared CF mutation test should be used and followed by bi‑directional sequencing when recommended by the test instructions for use.

Exclusion implicationPatients without a CFTR mutation responsive to ivacaftor per prescribing information or clinical/in‑vitro data are excluded from coverage under these criteria.

Background

Ivacaftor (Kalydeco) is a CFTR potentiator indicated for the treatment of cystic fibrosis in patients 1 month and older who have at least one CFTR mutation that is responsive to ivacaftor 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 and followed by bi‑directional sequencing when recommended by the test instructions. Members must meet the policy’s coverage criteria, including mutation confirmation, for prior authorization.