ModifiedHealth NetPolicy CP.PHAR.210

Clinical Policy: Ivacaftor (Kalydeco)

Defines medical necessity, approval criteria, dosing limits, and continuation requirements for ivacaftor (Kalydeco) for members covered by Health Net/Centene lines of business.

Policy Summary

PayerHealth Net

PolicyClinical Policy: Ivacaftor (Kalydeco)

Policy CodePolicy CP.PHAR.210

Change TypePediatric expansioncontinued therapy clarificationconcurrent exclusion added

Effective Date05.01.16

Next Review Date

Key ActionSubmit prior authorization with documentation confirming CF diagnosis, age, mutation responsive to ivacaftor, specialty prescribing, and age/weight‑appropriate dosing.

SourceLink

POLICY UPDATE CHANGES

Added pediatric expansion and new 5.8 mg and 13.4 mg granule strengths.

Revised continued therapy criteria language to 'stabilization or improvement in ppFEV1' and removed specific LCI thresholds.

Added Alyftrek to list of CFTR modulator concurrent exclusion criteria.

Age ≥ 1 monthminimum eligible age

300 mg/daymaximum adult daily dose

ppFEV1 within 90dbaseline spirometry (≥6 yrs)

Not with other CFTR modulatorsconcurrent therapy exclusion

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6 monthsinitial approval duration

5oral granule strengths

Coverage Criteria for Ivacaftor (Kalydeco)

Continued Therapy

Covered when ALL of the following are met:

ALL of the following

- ANY of the following
  - Currently receiving medication via Centene benefit or member has previously met initial approval criteria.
  - Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B).
- ANY of the following
  - For age < 6 years: Member is responding positively to therapy (e.g., decreased number of pulmonary exacerbations, increase in body mass index (BMI), improvement in respiratory symptoms).
  - For age ≥ 6 years: Member is responding positively to therapy as evidenced by stabilization or improvement (e.g., increase) in ppFEV1 from baseline.
Kalydeco is not prescribed concurrently with other CFTR modulators (e.g., Alyftrek, Orkambi, Symdeko, Trikafta).
Concurrent use with listed CFTR modulators is excluded.
- ANY of the following
  - Age 1 month to < 2 months and ≥ 3 kg: 11.6 mg per day (2 packets per day).
  - Age 2 months to < 4 months and ≥ 3 kg: 26.8 mg per day (2 packets per day).
  - Age 4 months to < 6 months and weight ≥ 5 kg: 50 mg per day (2 packets per day).
  - Age 6 months to < 6 years and weight 5 kg to < 7 kg: 50 mg per day (2 packets per day).
  - Age 6 months to < 6 years and weight 7 kg to < 14 kg: 100 mg per day (2 packets per day).
  - Age 6 months to < 6 years and weight ≥ 14 kg: 150 mg per day (2 packets per day).

Product Strengths, Dosing, and Coding

Product strengths (as listed)NDC | mixed

150 mg	Tablet strength listed in product availability
5.8 mg	Unit-dose packet oral granules
13.4 mg	Unit-dose packet oral granules
25 mg	Unit-dose packet oral granules
50 mg	Unit-dose packet oral granules
75 mg	Unit-dose packet oral granules

Sweat chloride — diagnostic threshold

Diagnostic sweat chloride thresholdElevated sweat chloride ≥ 60 mmol/L is required as evidence of CFTR dysfunction for CF diagnosis.

Maximum daily dose — age ≥6 years and general limits

Age ≥ 6 years maximum daily dose300 mg/day (one 150 mg tablet every 12 hours).

General dosing limitsDosing and administration vary by age and weight; follow age/weight-specific packet or tablet dosing per dosing table.

Approval duration noteApproval duration for initial therapy: 6 months (ensure dose does not exceed age-based maxima).

Prior Authorization, Documentation, and Denial Triggers

Prior Authorization

Prior Authorization Required

Prior authorization required. Provider must submit documentation (such as office chart notes, laboratory results, pulmonary function test reports, genetic testing, or other clinical information) that supports the member meets ALL applicable approval criteria. Prior authorization requests should indicate the specialty prescriber (pulmonologist) or documentation of consultation with a pulmonologist when applicable.

Prior authorization required for all requests.
Prescriber expectation: request should be submitted by or in consultation with a pulmonologist for cystic fibrosis indications.

Prior Authorization

Prior Authorization — Pediatric Expansion and Approval Duration

Pediatric expansion and labeling changes have been incorporated. Prior authorization requests for newly eligible pediatric ages or new granule strengths must include documentation appropriate to the expanded indication and dosing. Initial approval duration has been standardized to 6 months (consolidated from legacy durations).

If request reflects a recent label/age expansion not yet reflected in local formularies, reference off-formulary/no-coverage guidance per business line (see policy block B.1).

Background and Indications

Ivacaftor is a CFTR potentiator approved for use in cystic fibrosis patients with at least one ivacaftor‑responsive CFTR mutation. The agent improves CFTR channel gating and is indicated for patients aged 1 month and older who meet diagnostic criteria for CF and have an appropriate responsive mutation. Clinical confirmation of CF should include compatible symptoms or newborn screening and evidence of CFTR dysfunction (for example, sweat chloride ≥ 60 mmol/L or two disease‑causing CFTR mutations).

Definitions and References

ppFEV1 definition and use

ppFEV1Percent predicted forced expiratory volume in 1 second (ppFEV1).

Use in criteriaBaseline ppFEV1 required for age ≥ 6 years within last 90 days to document baseline lung function.

Continued therapyContinued approval may require stabilization or improvement in ppFEV1 compared to baseline.

CFTR — definition and relevance

CFTRCystic fibrosis transmembrane conductance regulator (CFTR).

Role in diagnosisEvidence of CFTR dysfunction (e.g., sweat chloride ≥ 60 mmol/L or two disease-causing CFTR mutations) is required to confirm CF diagnosis.

Policy Revision History

05.04.23RT4 pediatric expansion

RT4 revision expanded criteria to include pediatric population and added new 5.8 mg and 13.4 mg granule strengths.

06.23P&T approval

RT4 revision (pediatric expansion and new granule strengths) received P&T committee approval in June 2023.

05.10.23Annual review (3Q23)

3Q23 annual review noted no significant changes following the comprehensive RT4 review completed in June 2023.

Policy Summary

PayerHealth Net

PolicyClinical Policy: Ivacaftor (Kalydeco)

Policy CodePolicy CP.PHAR.210

Change TypePediatric expansioncontinued therapy clarificationconcurrent exclusion added

Effective Date05.01.16

Next Review Date

Key ActionSubmit prior authorization with documentation confirming CF diagnosis, age, mutation responsive to ivacaftor, specialty prescribing, and age/weight‑appropriate dosing.

SourceLink

On This Page

Age ≥ 6 years: 300 mg per day (2 tablets per day).

Maximum daily dose — age/weight-specific limits

Age 1 month to <2 months (weight ≥3 kg)Maximum 11.6–11.8 mg/day (2 packets per day).

Age 2 months to <4 months (weight ≥3 kg)Maximum 26.8 mg/day (2 packets per day).

Age 4 months to <6 months (weight ≥5 kg)Maximum 50 mg/day (2 packets per day).

Age 6 months to <6 years by weight5–<7 kg: 50 mg/day; 7–<14 kg: 100 mg/day; ≥14 kg: 150 mg/day (all given as two packets per day).

Age ≥6 years (including adults)Maximum 300 mg/day (two 150 mg tablets per day).

Standard initial approval duration: 6 months.

Documentation Required

Clinical Response Documentation Required for Continued Therapy

For continuation of therapy, submit documentation demonstrating clinical response compared to baseline. Continued coverage requires evidence of stabilization or improvement in clinically relevant measures (for example, increase or stabilization in percent predicted FEV1 compared to baseline).

Continued therapy requires documentation showing stabilization or improvement in ppFEV1 from baseline.
Baseline ppFEV1 must be clearly identified so follow-up comparisons are possible.

Step Therapy

Policy Sequencing / Step Guidance

If the requested use, age, or dosing is not listed in the policy or the policy is outdated due to recent label changes, follow the sequencing guidance: first determine if the drug is on-formulary or off-formulary and follow the relevant no-coverage or non-formulary policy for the line of business; if not covered by those, refer to the off-label use policy for the line of business.

For formulary drugs with label changes: consult the no coverage criteria policy for the relevant line of business (CP.CPA.190 commercial, HIM.PA.33 marketplace, CP.PMN.255 Medicaid).
For drugs not on the formulary: consult the non-formulary policy (CP.CPA.190 commercial, HIM.PA.103 marketplace, CP.PMN.16 Medicaid).
If use is not listed and criterion 1 does not apply: consult off-label use policy (CP.CPA.09 commercial, HIM.PA.154 marketplace, CP.PMN.53 Medicaid).

Documentation Required

Required Documentation — What to Submit

Required documentation to include with prior authorization: office chart notes, genetic testing results (confirming CFTR mutations and presence of an ivacaftor‑responsive mutation), sweat chloride results if available, baseline pulmonary function test results (see baseline spirometry callout), and dosing rationale. Absence of these documents may delay or prevent approval.

Acceptable documentation: office notes, lab reports, genetic test reports, sweat chloride test results, pulmonary function test reports, and specialist consultation notes.
Documentation must clearly demonstrate the member meets diagnostic criteria (clinical symptoms, CFTR dysfunction, presence of an ivacaftor‑responsive mutation, and absence of homozygous F508del).

Documentation Required

Baseline Spirometry Requirement

Baseline spirometry requirement: for members age ≥ 6 years, include percent predicted FEV1 (ppFEV1) performed within 90 days prior to the request to establish baseline lung function for initial and continued therapy assessment.

Baseline ppFEV1 must be within 90 days of the prior authorization request for members ≥ 6 years.
If ppFEV1 is not available and the member is ≥ 6 years, provide explanation and any alternative pulmonary function test documentation.

Denial Risk

Denial Triggers — What May Lead to Non‑Coverage

Denial triggers include missing or insufficient diagnostic documentation, failure to demonstrate an ivacaftor‑responsive CFTR mutation, concurrent use with excluded CFTR modulators, dosing in excess of policy limits, or lack of specialty prescriber/consultation when required.

Missing diagnostic criteria (clinical symptoms, evidence of CFTR dysfunction, or genetic confirmation) may lead to denial.
Lack of documentation showing presence of an ivacaftor‑responsive mutation is a denial trigger.
Requests exceeding the policy dose limits will be denied.
Concurrent use with excluded CFTR modulators (see concurrent exclusion callout) may result in denial.

Denial Risk

Concurrent CFTR Modulator Exclusion

Concurrent use exclusion: Kalydeco (ivacaftor) is not covered when prescribed concurrently with other CFTR modulators including Alyftrek, Orkambi, Symdeko, or Trikafta. Documented concurrent therapy with any excluded CFTR modulator may result in coverage denial.

Concurrent administration with Alyftrek, Orkambi, Symdeko, or Trikafta is excluded.
If clinical rationale for combination therapy is provided, include supporting evidence and justification — however concurrent use is generally a denial trigger.

Mutation responsivenessPresence of at least one CFTR mutation responsive to ivacaftor is required for therapy eligibility; homozygous F508del must be absent.

References — prescribing information and clinical guidelines

Kalydeco Prescribing InformationKalydeco (ivacaftor) Prescribing Information — Vertex Pharmaceuticals; referenced Aug 2023 (accessed Apr 14, 2025).

Cystic Fibrosis Foundation guidelinesCystic Fibrosis Foundation clinical care and pulmonary guidelines (e.g., 2013 chronic medications; 2018 CFTR modulator guidance).

Diagnosis consensusDiagnosis of cystic fibrosis: Consensus guidelines from the Cystic Fibrosis Foundation (Farrell et al., 2017).

02.22P&T approval (earlier template changes)

Template and reference updates were applied with P&T committee approval in February 2022.

10.06.221Q2023 annual review

Consolidated legacy Wellcare initial approval duration from 12 months to 6 months and updated references and appendices.

05.04.23Added granule strengths

Documented addition of 5.8 mg and 13.4 mg unit-dose oral granule strengths as part of the RT4 update.

06.23Alyftrek addition (concurrent exclusion)Latest

Alyftrek was later added to the list of excluded concurrent CFTR modulators as part of subsequent policy updates.