ModifiedCentenePolicy CP.PHAR.210

Ivacaftor (Kalydeco)

Medical necessity and prior authorization criteria for ivacaftor (Kalydeco) for treatment of cystic fibrosis for Centene-affiliated health plans, covering commercial, HIM, and Medicaid lines of business.

Policy Summary

PayerCentene

PolicyIvacaftor (Kalydeco) coverage criteria

Policy CodePolicy CP.PHAR.210

Change TypePediatric expansiondosing/formulation additionsexclusion list update

Effective DateMay 1, 2016

Next Review Date

Key ActionSubmit prior authorization with documentation of CF diagnosis, genotype confirming an ivacaftor‑responsive mutation, age, prescriber specialty, and required baseline/response pulmonary function testing.

SourceLink

POLICY UPDATE CHANGES

Added 5.8 mg and 13.4 mg granule strengths and expanded pediatric dosing to include younger age groups.

Revised continued therapy criteria language to require 'stabilization or improvement in ppFEV1' and removed lung clearance index (LCI) from criteria.

Added Alyftrek to the list of CFTR modulator concurrent exclusion criteria.

≥1 monthminimum approved patient age

6 monthsinitial approval length

Within 90 daysppFEV1 baseline timing

Not with other CFTR modulatorsconcurrent therapy exclusion

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage Criteria

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial Approval Criteria - Cystic Fibrosis: 1. Diagnosis of cystic fibrosis (CF) confirmed by all of the following (a, b, c, and d): a. Clinical features consistent with CF in at least one organ system, or positive newborn screen or genetic testing for siblings of patients with CF; b. Evidence of CFTR dysfunction confirmed by either i. elevated sweat chloride ≥ 60 mmol/L OR ii. genetic testing confirming two disease‑causing CFTR mutations (one from each parental allele); c. Presence of one CFTR mutation responsive to ivacaftor based on clinical and/or in vitro assay data; d. Confirmation that a homozygous F508del mutation is not present; 2. Age ≥ 1 month; 3. Medication is prescribed by or in consultation with a pulmonologist; 4. For members age ≥ 6 years: documentation of baseline percent predicted FEV1 (ppFEV1) performed within the last 90 days; 5. Ivacaftor (Kalydeco) is not prescribed concurrently with other CFTR modulators (e.g., Alyftrek, Orkambi, Symdeko, Trikafta); 6. Dose does not exceed the age‑ and weight‑based maximums specified in the dosing table (see policy dosing).

inv-02: Continuation Therapy

Covered for continuation when ALL of the following are met:

Continued Therapy - Cystic Fibrosis: 1. Member meets one of the following: a. Currently receiving ivacaftor via the Centene benefit or previously met initial approval criteria; OR b. Member is currently receiving medication and is in a continuity‑of‑care situation per applicable state/product rules; 2. Member is responding positively to therapy as follows: a. For age < 6 years: clinical response (e.g., decreased pulmonary exacerbations, increased BMI, improved respiratory symptoms); b. For age ≥ 6 years: response evidenced by stabilization or improvement in ppFEV1 from baseline; 3. Ivacaftor is not prescribed concurrently with other CFTR modulators (e.g., Alyftrek, Orkambi, Symdeko, Trikafta); 4. If a dose increase is requested, the new dose must not exceed the age‑ and weight‑based maximums specified in the dosing table;

inv-03: Initial and Continued Therapy Criteria

Coverage requires meeting dosing, indication, and response documentation as specified.

Dosing by age/weight: Patient must be prescribed ivacaftor at an age‑ and weight‑appropriate dose and formulation per the policy dosing table.see dosing thresholds

Available formulations include oral granules (5.8 mg, 13.4 mg, 25 mg, 50 mg, 75 mg) and 150 mg tablet; maximum daily doses vary by age/weight (e.g., age ≥ 6 years: 300 mg/day).

Continued therapy response: For continued authorization, documentation must demonstrate stabilization or improvement in ppFEV1 from baseline.stabilization or improvement from baseline

Policy removed lung clearance index (LCI) from required response metrics; documentation of pulmonary function testing per policy is required.

Concurrent therapy exclusion: Therapy is not covered when prescribed concurrently with excluded CFTR modulators.

Non‑FDA approved indications that are not specifically addressed in this policy are not covered unless the provider supplies sufficient documentation of safety and efficacy consistent with the applicable off‑label use policies. Follow the referenced off‑label policies: CP.CPA.09 (Commercial), HIM.PA.154 (Health Insurance Marketplace), and CP.PMN.53 (Medicaid) when submitting requests for indications outside the FDA‑approved label.

Ivacaftor is not covered when prescribed concurrently with other CFTR modulators listed as exclusions in this policy. The concurrent‑use exclusion list was recently expanded to include Alyftrek; prior or concomitant therapy with excluded CFTR modulators may prevent approval.

Kalydeco (ivacaftor) is not effective for patients who are homozygous for the F508del CFTR mutation; use in this population is not supported by the policy and will not meet coverage criteria.

This policy applies to Centene lines of business including Medicaid; however, state Medicaid coverage provisions take precedence when they conflict with this clinical policy. Refer to the applicable state Medicaid manual for coverage rules. For non‑FDA indications in any line of business, follow the off‑label policies referenced in this document.

Product Availability and Dosing / Coding

NDCNDC

00093-3200-01	Ivacaftor 150 mg tablet (example NDC - verify product-specific NDC)
00093-3201-01	Ivacaftor oral granules 5.8 mg packet (56/carton) (example NDC - verify)
00093-3202-01	Ivacaftor oral granules 13.4 mg packet (56/carton) (example NDC - verify)
00093-3203-01	Ivacaftor oral granules 25 mg packet (56/carton) (example NDC - verify)
00093-3204-01	Ivacaftor oral granules 50 mg packet (56/carton) (example NDC - verify)
00093-3205-01	Ivacaftor oral granules 75 mg packet (56/carton) (example NDC - verify)

Baseline ppFEV1 Timing — Patients ≥ 6 years

Baseline ppFEV1 timing (age ≥ 6 years)Documentation of percent predicted FEV1 (ppFEV1) performed within the last 90 days is required.

Maximum Daily Dose by Age Group

Maximum daily dose — Age 1 month to <2 months (weight ≥3 kg)11.6–11.8 mg/day (one 5.8 mg packet PO every 12 hours)

Maximum daily dose — Age 2 months to <4 months (weight ≥3 kg)26.8 mg/day (one 13.4 mg packet PO every 12 hours)

Maximum daily dose — Age 4 months to <6 months (weight ≥5 kg)50 mg/day (one 25 mg packet PO every 12 hours)

Maximum daily dose — Age 6 months to <6 years (weight 5 kg to <7 kg)50 mg/day (one 25 mg packet PO every 12 hours)

Provider Actions and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Provider must submit documentation (office chart notes, lab results, genetic testing, pulmonary function tests, and other clinical information) supporting that the member meets all approval criteria.

Diagnosis documentation: clinical symptoms, newborn screen or genetic testing for siblings of CF patients.
CFTR dysfunction evidence: elevated sweat chloride ≥ 60 mmol/L OR genetic testing showing two disease-causing CFTR mutations (one from each parental allele).
Mutation responsive to ivacaftor: presence of at least one CFTR mutation responsive to ivacaftor per Appendix E.
Exclude homozygous F508del: confirmation that member is not homozygous for F508del.
Age and prescriber: age ≥ 1 month and prescribed by or in consultation with a pulmonologist.
Baseline testing for age ≥ 6 years: ppFEV1 performed within the last 90 days.
Approval duration: initial approval up to 6 months.

Background

Ivacaftor is a CFTR potentiator indicated for treatment of cystic fibrosis in patients with at least one ivacaftor‑responsive CFTR mutation. The drug increases activity of the CFTR protein at the cell surface (potentiation), which can improve chloride transport in responsive mutations. Clinical use requires confirmation of CF diagnosis and genotype, and the policy specifies that homozygous F508del patients are not considered appropriate candidates for ivacaftor therapy.

Definitions

ppFEV1 (abbreviation)

AbbreviationppFEV1

MeaningPercent predicted forced expiratory volume in 1 second

Location in policyDefined in Appendix A: Abbreviation/Acronym Key

CFTR (abbreviation)

AbbreviationCFTR

MeaningCystic fibrosis transmembrane conductance regulator

ContextCFTR mutations determine responsiveness to ivacaftor per policy requirements

Policy Summary

PayerCentene

PolicyIvacaftor (Kalydeco) coverage criteria

Policy CodePolicy CP.PHAR.210

Change TypePediatric expansiondosing/formulation additionsexclusion list update

Effective DateMay 1, 2016

Next Review Date

SourceLink

On This Page

concurrent use with listed CFTR modulators

Alyftrek was added to the list of excluded CFTR modulators; see exclusions list for full details.

Maximum daily dose — Age 6 months to <6 years (weight 7 kg to <14 kg)100 mg/day (one 50 mg packet PO every 12 hours)

Maximum daily dose — Age 6 months to <6 years (weight ≥14 kg)150 mg/day (one 75 mg packet PO every 12 hours)

Maximum daily dose — Age ≥6 years300 mg/day (one 150 mg tablet PO every 12 hours)

Formulary / Off‑Label Pathway

When requested use is not listed in the diagnoses/indications or the policy is not up to date after a recent label change, follow the applicable formulary or off‑label pathways for the member's line of business.

If the drug recently underwent a label change not yet reflected in this policy: follow formulary/non‑formulary policy for the applicable line of business (commercial, marketplace, Medicaid) as referenced.
If the requested use is not listed under authorized indications and no recent label change applies: refer to the off‑label use policy for the relevant line of business (commercial, marketplace, Medicaid).

Denial Risk

Concurrent CFTR Modulator Exclusion

Concurrent use with other CFTR modulators is excluded. Document prior therapy and ensure no overlapping CFTR modulators are prescribed.

Kalydeco (ivacaftor) must not be prescribed concurrently with other CFTR modulators (examples include Alyftrek®, Orkambi®, Symdeko®, Trikafta™).
Alyftrek was added to the concurrent‑use exclusion in 3Q 2025 — verify current medication list and prior therapies before approval.
Prior therapy with a CFTR modulator may affect eligibility — submit medication history.
Medicaid precedence: when state Medicaid provisions conflict with this policy, state rules take precedence.

ppFEV1 — definition and clinical use

DefinitionPercent predicted forced expiratory volume in 1 second (ppFEV1) — a measure of lung function expressed as percent of predicted normal for age/sex/height.

Use in policyUsed to document baseline lung function and to demonstrate stabilization or improvement for continued therapy (policy requires stabilization or improvement from baseline for age ≥ 6 years).

DocumentationAcceptable documentation includes pulmonary function test reports referenced in continued-therapy criteria and dosing sections.

LCI — lung clearance index (definition and removal)

DefinitionLCI (lung clearance index) — a multiple-breath washout metric previously used as a response measure.

Policy changeLCI was removed from the continued therapy criteria to align with standard of care (removed 12.31.24; reflected in 2025 review).

ImplicationLCI is no longer required or accepted as a primary response metric for continued authorization under this policy; ppFEV1 is the required metric for age ≥ 6 years.