CurrentUnitedHealthcarePolicy N/A

Orkambi (lumacaftor/ivacaftor) prior authorization

Prior authorization and medical necessity criteria for coverage of Orkambi (lumacaftor/ivacaftor) for treatment of cystic fibrosis in eligible members; applies to UnitedHealthcare pharmacy benefit management programs.

Policy Summary

PayerUnitedHealthcare

PolicyOrkambi (lumacaftor/ivacaftor) prior authorization

Policy CodePolicy N/A

Change TypeNo material change

Effective DateSep 1, 2025

Next Review DateJun 1, 2025

Key ActionObtain prior authorization with documentation of CF diagnosis, patient age ≥ 1 year, and laboratory confirmation of homozygous F508del mutation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥1minimum patient age for coverage

homozygous F508delrequired CFTR genotype

12 moauthorization duration

Prior Authprogram type

Coverage Criteria for Orkambi (lumacaftor/ivacaftor)

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Initial Authorization: a. Diagnosis of cystic fibrosis (CF)

Initial Authorization: b. Submission of laboratory results confirming that patient is homozygous for the F508del mutation in the CFTR gene

Initial Authorization: c. The patient is ≥ 1 years of age

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

Authorization will be issued for 12 months.

Reauthorization

Reauthorization — Covered when the following is met:

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

Authorization will be issued for 12 months.

Use of Orkambi in patients with cystic fibrosis who are not homozygous for the F508del mutation is not established. Limitations of use in the source state that the efficacy and safety of Orkambi have not been established for patients with CF other than those homozygous for F508del; therefore such genotypes are excluded from coverage criteria.

Initial Therapy

Initial coverage criteria

Initial therapy: Diagnosis of CF

Initial therapy: Age ≥ 1 year

Initial therapy: Laboratory confirmation of homozygous F508del mutation in the CFTR gene

Initial therapy: Prescribed by or in consultation with a CF specialist

12-month authorization.

Reauthorization / Continuation Therapy

Reauthorization requirements for continuation of therapy

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

Authorization renewed for 12 months.

Provider Actions and Prior Authorization Requirements

Prior Authorization

Orkambi prior authorization requirements

Prior authorization for Orkambi must include documentation of a cystic fibrosis diagnosis, patient age ≥ 1 year, laboratory confirmation that the patient is homozygous for the F508del mutation in the CFTR gene, and that the prescription is by or in consultation with a provider who specializes in the treatment of cystic fibrosis. Approvals (initial and reauthorization) are issued for 12 months.

Diagnosis of cystic fibrosis (CF)
Laboratory results confirming homozygosity for F508del
Patient is ≥ 1 year of age
Prescribed by or in consultation with a CF specialist
Authorization duration: 12 months

Step Therapy

Automated approval and supply limits

UnitedHealthcare may use automated approval and reauthorization processes based on prior claim/medication history and diagnosis codes; programs may apply supply limits.

Automated approval/re-approval may use previous claim or medication history and ICD-10 diagnosis codes
Use of automated processes varies by program/therapeutic class
Supply limits may apply

Documentation Required

Required documentation

Submit laboratory results that confirm the patient is homozygous for the F508del mutation on both CFTR alleles, documentation of cystic fibrosis diagnosis, and the patient's age to support authorization.

If genotype is unknown, use an FDA-cleared CF mutation test to detect F508del on both alleles
Include patient age and CF diagnosis documentation

Denial Risk

Genotype and diagnosis requirement

Coverage may be denied when the patient is not homozygous for the F508del mutation or when the diagnosis is not cystic fibrosis because efficacy and safety have not been established for other genotypes.

Efficacy/safety not established for CF genotypes other than homozygous F508del
Members must meet the coverage criteria to obtain approval

Background

Orkambi (lumacaftor/ivacaftor) is a combination CFTR potentiator therapy indicated for treatment of cystic fibrosis in patients aged ≥ 1 year who are homozygous for the F508del mutation. If the patient’s genotype is unknown, an FDA‑cleared CF mutation test should be used to confirm presence of F508del on both alleles. The efficacy and safety of Orkambi have not been established for patients with CF who are not homozygous for F508del.

Definitions

Homozygous F508del (definition & policy implications)

DefinitionHomozygous for the F508del mutation: presence of the F508del mutation on both CFTR gene alleles as confirmed by an FDA-cleared CF mutation test.

Relevance to coverageOrkambi is indicated only for patients aged ≥ 1 year who are homozygous for F508del; efficacy and safety are not established for other genotypes.

Testing guidanceIf genotype is unknown, use an FDA-cleared CF mutation test to detect F508del on both alleles before authorization.

Age requirementApplies to patients aged 1 year and older who meet homozygous F508del definition.

Documentation requirementLaboratory results confirming homozygosity for F508del must be provided with the prior authorization request.