Drug · FAERS side-effect profile with label screen
Ivacaftor side effects: what 7,893 FDA adverse event reports show
Drug class
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
FDA label (Established Pharmacologic Class) · compare the class
Brand names
Kalydeco
openFDA label
Who reports
7,893 reports · 74% serious · 325 with death outcome · 57% female
openFDA FAERS counts, all years, deduplication not applied
Used for
KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data .
FDA label, Indications and Usage
What it is
The active ingredient in KALYDECO tablets and oral granules is ivacaftor, a cystic fibrosis transmembrane conductance regulator potentiator, which has the following chemical name: N -(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide.
FDA label, Description section
Boxed warning
None in the current label
How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation.
Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)?
That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.
Causality screen of the 40 strongest signals
Labeled (related wording) 3Likely indication confounding 3Reporting / coding artifact 4Lab value / disease marker 4Not in label — unassessed 26
What the badges mean
- Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
- Likely indication confounding The term appears in the label's indications: reports probably describe the disease being treated (or its progression), not a drug effect. Classic confounding by indication.
- Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
- Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
- Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.
Strongest disproportionality signals
Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 653 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.
Show:
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Amniotic fluid index increased | 15 | 13,096.6 3,792.2–45,230.4ROR 13,121.5 · χ² 30,590 / p <1e-300 | 574.4 EB05 369.5 · IC025 +4.1 3/3 methods agree | Lab value / disease marker | serious n=14 | no trial data cached |
| Sweat test abnormal | 16 | 891.7 505.8–1,571.8ROR 893.5 · χ² 9,965 / p <1e-300 | 370.2 EB05 241.6 · IC025 +4.1 3/3 methods agree | Reporting / coding artifact | serious n=7 | no trial data cached |
| Infective pulmonary exacerbation of cystic fibrosis | 610 | 332.7 306.7–360.8ROR 360.4 · χ² 178,729 / p <1e-300 | 292.6 EB05 273.6 · IC025 +7.8 3/3 methods agree | Likely indication confounding shares a disease stem with the indications wording | serious n=520 | no trial data cached |
| Cystic fibrosis related diabetes | 12 | 239.9 132.9–433.2ROR 240.3 · χ² 2,401 / p <1e-300 | 163.3 EB05 99.5 · IC025 +3.5 3/3 methods agree | Likely indication confounding shares a disease stem with the indications wording | serious n=8 | no trial data cached |
| Cystic fibrosis respiratory infection suppression | 73 | 234.6 184.8–297.8ROR 236.8 · χ² 15,372 / p <1e-300 | 204.0 EB05 167.6 · IC025 +6.1 3/3 methods agree | Labeled (related wording) related wording | serious n=72 | no trial data cached |
| Distal intestinal obstruction syndrome | 27 | 228.9 154.5–339.0ROR 229.7 · χ² 5,426 / p <1e-300 | 183.4 EB05 132.4 · IC025 +4.8 3/3 methods agree | Not in label — unassessed | serious n=27 | no trial data cached |
| Foetal macrosomia | 16 | 165.0 99.6–273.3ROR 165.3 · χ² 2,302 / p <1e-300 | 131.3 EB05 85.7 · IC025 +3.9 3/3 methods agree | Not in label — unassessed | serious n=16 | no trial data cached |
| Left-to-right cardiac shunt | 15 | 141.3 84.1–237.5ROR 141.6 · χ² 1,852 / p <1e-300 | 114.9 EB05 73.9 · IC025 +3.8 3/3 methods agree | Not in label — unassessed | serious n=15 | no trial data cached |
| Cystic fibrosis | 218 | 108.3 94.7–123.8ROR 111.3 · χ² 22,156 / p <1e-300 | 103.1 EB05 92.1 · IC025 +6.2 3/3 methods agree | Likely indication confounding term in indications | serious n=207 | no trial data cached |
| Pulmonary function test decreased | 138 | 86.0 72.7–101.7ROR 87.5 · χ² 11,140 / p <1e-300 | 82.3 EB05 71.4 · IC025 +5.7 3/3 methods agree | Not in label — unassessed | serious n=109 · 70% consumer | no trial data cached |
| Chalazion | 14 | 59.9 35.3–101.7ROR 60.0 · χ² 736 / p 2e-162 | 54.5 EB05 34.5 · IC025 +3.4 3/3 methods agree | Not in label — unassessed | serious n=14 | no trial data cached |
| Motor developmental delay | 12 | 56.4 31.9–99.9ROR 56.5 · χ² 586 / p 7e-130 | 51.1 EB05 31.1 · IC025 +3.1 3/3 methods agree | Not in label — unassessed | serious n=12 | no trial data cached |
| Forced expiratory volume decreased | 45 | 42.8 31.9–57.4ROR 43.0 · χ² 1,766 / p <1e-300 | 41.4 EB05 32.2 · IC025 +4.4 3/3 methods agree | Lab value / disease marker | serious n=37 · ♀ 31% (drug 57%) | no trial data cached |
| Oesophageal achalasia | 10 | 42.0 22.5–78.5ROR 42.1 · χ² 355 / p 2e-79 | 38.6 EB05 22.4 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious n=10 | no trial data cached |
| Bronchial secretion retention | 19 | 40.5 25.8–63.7ROR 40.6 · χ² 682 / p 1e-150 | 38.5 EB05 26.0 · IC025 +3.5 3/3 methods agree | Not in label — unassessed | serious n=15 | no trial data cached |
| Sputum increased | 30 | 38.2 26.6–54.7ROR 38.3 · χ² 1,034 / p 3e-227 | 36.8 EB05 27.1 · IC025 +3.9 3/3 methods agree | Labeled (related wording) related wording | serious n=24 | no trial data cached |
| Intensive care | 19 | 36.6 23.3–57.6ROR 36.7 · χ² 615 / p 4e-136 | 35.0 EB05 23.7 · IC025 +3.5 3/3 methods agree | Not in label — unassessed | serious n=19 | no trial data cached |
| Pseudomonas test positive | 11 | 35.9 19.8–65.0ROR 35.9 · χ² 334 / p 5e-75 | 33.5 EB05 19.9 · IC025 +2.8 3/3 methods agree | Lab value / disease marker | serious n=5 | no trial data cached |
| Butterfly rash | 12 | 32.2 18.2–56.8ROR 32.2 · χ² 328 / p 1e-73 | 30.4 EB05 18.5 · IC025 +2.9 3/3 methods agree | Not in label — unassessed | serious n=12 | no trial data cached |
| Lung transplant | 42 | 31.6 23.4–42.9ROR 31.8 · χ² 1,201 / p 1e-263 | 30.9 EB05 23.8 · IC025 +4.0 3/3 methods agree | Not in label — unassessed | serious n=42 | no trial data cached |
| Hospitalisation | 1,357 | 23.9 22.7–25.0ROR 28.6 · χ² 29,637 / p <1e-300 | 23.6 EB05 22.6 · IC025 +4.5 3/3 methods agree | Reporting / coding artifact | serious PRR 18.7 · 88% consumer | no trial data cached |
| Pneumonia pseudomonal | 13 | 22.9 13.3–39.5ROR 22.9 · χ² 249 / p 3e-56 | 21.9 EB05 13.4 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious n=13 | no trial data cached |
| Sinus operation | 18 | 20.3 12.7–32.2ROR 20.3 · χ² 308 / p 3e-69 | 19.6 EB05 12.9 · IC025 +2.9 3/3 methods agree | Reporting / coding artifact | serious n=16 | no trial data cached |
| Pseudomonas infection | 64 | 20.2 15.8–25.8ROR 20.4 · χ² 1,142 / p 1e-250 | 20.0 EB05 16.2 · IC025 +3.7 3/3 methods agree | Not in label — unassessed | serious PRR 10.7 | no trial data cached |
| Increased bronchial secretion | 17 | 17.7 11.0–28.5ROR 17.7 · χ² 250 / p 1e-56 | 17.0 EB05 10.9 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious n=11 | no trial data cached |
| Weight gain poor | 14 | 16.2 9.6–27.3ROR 16.2 · χ² 183 / p 5e-42 | 15.2 EB05 8.9 · IC025 +2.5 3/3 methods agree | Not in label — unassessed | serious n=6 | no trial data cached |
| Lower respiratory tract infection | 251 | 14.8 13.1–16.7ROR 15.2 · χ² 3,201 / p <1e-300 | 14.7 EB05 13.2 · IC025 +3.6 3/3 methods agree | Labeled (related wording) related wording | serious PRR 11.9 · 95% consumer | no trial data cached |
| Infectious mononucleosis | 11 | 13.0 7.2–23.5ROR 13.0 · χ² 110 / p 6e-26 | 11.2 EB05 5.9 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | serious n=6 | no trial data cached |
| Pulmonary haemorrhage | 37 | 12.6 9.1–17.4ROR 12.7 · χ² 382 / p 2e-85 | 12.2 EB05 9.0 · IC025 +2.9 3/3 methods agree | Not in label — unassessed | serious PRR 9.8 | no trial data cached |
| Staphylococcus test positive | 10 | 12.6 6.7–23.4ROR 12.6 · χ² 95 / p 1e-22 | 10.5 EB05 5.3 · IC025 +1.9 3/3 methods agree | Lab value / disease marker | serious n=7 | no trial data cached |
| Disturbance in social behaviour | 10 | 11.3 6.1–20.9ROR 11.3 · χ² 83 / p 4e-20 | 9.1 EB05 4.7 · IC025 +1.8 3/3 methods agree | Reporting / coding artifact | serious n=10 | no trial data cached |
| Transplant | 10 | 11.2 6.0–20.9ROR 11.2 · χ² 83 / p 5e-20 | 9.1 EB05 4.7 · IC025 +1.8 3/3 methods agree | Not in label — unassessed | serious n=10 | no trial data cached |
| Haemoptysis | 117 | 11.2 9.3–13.4ROR 11.3 · χ² 1,068 / p 1e-234 | 11.0 EB05 9.4 · IC025 +3.1 3/3 methods agree | Not in label — unassessed | serious PRR 7.8 | no trial data cached |
| Speech disorder developmental | 12 | 11.1 6.3–19.5ROR 11.1 · χ² 100 / p 8e-24 | 9.3 EB05 5.2 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | serious n=12 | no trial data cached |
| Pneumonia viral | 12 | 10.0 5.7–17.7ROR 10.0 · χ² 88 / p 3e-21 | 8.3 EB05 4.7 · IC025 +1.9 3/3 methods agree | Not in label — unassessed | serious n=11 | no trial data cached |
| Hepatic necrosis | 14 | 9.9 5.8–16.7ROR 9.9 · χ² 102 / p 2e-24 | 8.3 EB05 5.0 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | serious n=14 | no trial data cached |
| Behaviour disorder | 18 | 9.0 5.7–14.3ROR 9.1 · χ² 120 / p 3e-28 | 7.9 EB05 5.1 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | serious n=17 | no trial data cached |
| Emergency care | 10 | 9.0 4.8–16.7ROR 9.0 · χ² 63 / p 1e-15 | 7.0 EB05 3.8 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious n=2 | no trial data cached |
| Lung infection | 51 | 8.6 6.6–11.3ROR 8.7 · χ² 335 / p 4e-75 | 8.2 EB05 6.4 · IC025 +2.5 3/3 methods agree | Not in label — unassessed | serious PRR 5.7 | no trial data cached |
| Hepatic cytolysis | 38 | 8.3 6.0–11.4ROR 8.3 · χ² 235 / p 2e-53 | 7.7 EB05 5.8 · IC025 +2.4 3/3 methods agree | Not in label — unassessed | serious PRR 6.4 · 100% clinician | no trial data cached |
Trial evidence (ClinicalTrials.gov)
Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).
Most frequently reported reactions
By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Hospitalisation | 1,357 | 23.9 22.7–25.0ROR 28.6 · χ² 29,637 / p <1e-300 | 23.6 EB05 22.6 · IC025 +4.5 3/3 methods agree | Reporting / coding artifact | serious PRR 18.7 · 88% consumer | no trial data cached |
| Infective pulmonary exacerbation of cystic fibrosis | 610 | 332.7 306.7–360.8ROR 360.4 · χ² 178,729 / p <1e-300 | 292.6 EB05 273.6 · IC025 +7.8 3/3 methods agree | Likely indication confounding shares a disease stem with the indications wording | serious n=520 | no trial data cached |
| Pneumonia (lung infection) | 269 | 2.2 2.0–2.5ROR 2.3 · χ² 180 / p 3e-41 | 2.2 EB05 2.0 · IC025 +0.9 | Labeled (related wording) related wording | serious PRR 1.6 | no trial data cached |
| Infection | 255 | 4.7 4.2–5.3ROR 4.8 · χ² 744 / p 5e-164 | 4.7 EB05 4.2 · IC025 +2.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 2.8 | no trial data cached |
| Malaise (feeling unwell) | 253 | 1.5 1.4–1.7ROR 1.5 · χ² 47 / p 4e-12 | 1.5 EB05 1.4 · IC025 +0.4 | Not in label — unassessed | serious PRR 1.4 · 74% consumer | no trial data cached |
| Lower respiratory tract infection | 251 | 14.8 13.1–16.7ROR 15.2 · χ² 3,201 / p <1e-300 | 14.7 EB05 13.2 · IC025 +3.6 3/3 methods agree | Labeled (related wording) related wording | serious PRR 11.9 · 95% consumer | no trial data cached |
| Death | 228 | 0.7 0.6–0.8ROR 0.7 · χ² 30 / p 3e-8 | 0.7 EB05 0.6 · IC025 -0.7 PRR-only: not supported by EBGM or IC | Outcome field, not a reaction | serious PRR 0.5 · 77% consumer | no trial data cached |
| Cough | 223 | 2.1 1.9–2.4ROR 2.1 · χ² 132 / p 7e-31 | 2.1 EB05 1.9 · IC025 +0.9 | Not in label — unassessed | serious PRR 1.1 | no trial data cached |
| Cystic fibrosis | 218 | 108.3 94.7–123.8ROR 111.3 · χ² 22,156 / p <1e-300 | 103.1 EB05 92.1 · IC025 +6.2 3/3 methods agree | Likely indication confounding term in indications | serious n=207 | no trial data cached |
| Headache | 196 | 0.8 0.7–1.0ROR 0.8 · χ² 7 / p 0.005 | 0.8 EB05 0.7 · IC025 -0.5 PRR-only: not supported by EBGM or IC | Labeled adverse reaction exact term | serious PRR 0.5 | no trial data cached |
| Nasopharyngitis (common cold) | 171 | 2.5 2.1–2.9ROR 2.5 · χ² 148 / p 3e-34 | 2.4 EB05 2.2 · IC025 +1.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 1.5 · 74% consumer | no trial data cached |
| Influenza | 161 | 3.8 3.3–4.5ROR 3.9 · χ² 335 / p 5e-75 | 3.8 EB05 3.3 · IC025 +1.7 3/3 methods agree | Not in label — unassessed | serious PRR 3.1 · 74% consumer | no trial data cached |
| Dyspnoea (shortness of breath) | 144 | 0.7 0.6–0.8ROR 0.7 · χ² 23 / p 1e-6 | 0.7 EB05 0.6 · IC025 -0.8 PRR-only: not supported by EBGM or IC | Not in label — unassessed | serious PRR 0.5 | no trial data cached |
| Pulmonary function test decreased | 138 | 86.0 72.7–101.7ROR 87.5 · χ² 11,140 / p <1e-300 | 82.3 EB05 71.4 · IC025 +5.7 3/3 methods agree | Not in label — unassessed | serious n=109 · 70% consumer | no trial data cached |
| Off label use (used outside approved label) | 124 | 0.4 0.3–0.5ROR 0.4 · χ² 131 / p 1e-30 | 0.4 EB05 0.3 · IC025 -1.7 PRR-only: not supported by EBGM or IC | Reporting / coding artifact | serious PRR 0.3 | no trial data cached |
Unlabeled candidate signals
These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.
- Distal intestinal obstruction syndrome — n=27, PRR 228.9
- Pulmonary function test decreased — n=138, PRR 86.0
- Lung transplant — n=42, PRR 31.6
- Pseudomonas infection — n=64, PRR 20.2
- Pulmonary haemorrhage — n=37, PRR 12.6
- Haemoptysis — n=117, PRR 11.2
- Lung infection — n=51, PRR 8.6
- Hepatic cytolysis — n=38, PRR 8.3
Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.
Common questions
What are the most reported side effects of Ivacaftor?
In FDA FAERS, the most frequently co-reported reaction terms for Ivacaftor are infective pulmonary exacerbation of cystic fibrosis, pneumonia, infection, malaise, lower respiratory tract infection, cough. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.
Does Ivacaftor have an FDA boxed warning?
No. The FDA label retrieved for Ivacaftor has no boxed warning; see the label's Warnings and Precautions for the recognised risks.
Do FAERS signals prove that Ivacaftor causes these side effects?
No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.
Sources & methods
- FDA label via openFDA (set ID 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25, effective 20260323; matched on
openfda.generic_name.exact:"IVACAFTOR") - Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
- FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA
count=aggregation, fieldpatient.drug.openfda.generic_name.exact, 693 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction - Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
- Seriousness and sex counts from openFDA
count=serious,patient.patientsex,seriousnessdeath
Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.