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Ivacaftor

  • Prescription medicine
  • Prescription only
  • Identity checked
  • Sources last checked 2026-09-04

This page shows what was measured, who it was measured in, and what that does not settle.

What Ivacaftor does in the body

Cystic fibrosis

From the FDA-approved label: Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel open probability (or gating) of CFTR protein located at the cell surface. The overall level of ivacaftor-mediated CFTR chloride transport is dependent on the amount of CFTR protein at the cell surface and how responsive a particular mutant CFTR protein is to ivacaftor potentiation.

What happened in people

RNAWiki has not yet published a reviewed conclusion for this use.

A fixed RNAWiki sentence

Where this came from

Wording RNAWiki always uses, not a finding about this substance.

No reviewed claim names a result for any goal on this record.

No source is stored against this line.

The limit that matters most

Not recorded.

Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

What the registries record it as

  • The substance registry classes this as chemical.

    FDA substance registry · 1Y740ILL1Z · read 2026-08-29

  • Its recorded molecular formula is C24H28N2O3, weighing 392.49.

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

Where each sentence above came from

No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.

Shown as the opening line on this page.

No statement of the main limit is recorded.

The four opening statements run to 97 words.

Words this page uses

Four words worth knowing first

Chosen from what this page shows, with each one explained before the word it depends on.

Biomarker

A biomarker is a number from a test that stands in for something about health.

A picture of it, and where the picture fails

A biomarker is like a fuel gauge.

Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.

What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.

A measurable indicator used as a substitute for a clinical outcome of interest.

Placebo

A placebo is a dummy treatment given so the real one can be compared with it.

A picture of it, and where the picture fails

A placebo is like a blank control in an experiment.

Where that stops being true. A blank does nothing. People given a placebo often do get better.

What people get wrong. A placebo effect is read as imaginary. The improvement is measured and real.

An inactive intervention matched in appearance to the test intervention, used to control for non-specific effects.

Comparator

A comparator is whatever the treatment was measured against.

A picture of it, and where the picture fails

It is like the other runner in a race.

Where that stops being true. A race has one winner. A study can show both arms improved.

What people get wrong. Results are read without asking what the other group got. Beating nothing is not beating a treatment.

The control condition against which the experimental intervention is assessed.

What happened in peopleRead from sources, not yet reviewed

What happened in people

Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.

Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

The study did not show it

Who was studied
NCT01931839
How many people
1164
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

The study did not show it

Who was studied
NCT02565914
How many people
1131
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1)

The study did not show it

Who was studied
NCT05076149
How many people
597
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24

The study did not show it

Who was studied
NCT01807949
How many people
563
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24

The study did not show it

Who was studied
NCT01807923
How many people
559
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot

Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24

The study did not show it

Who was studied
NCT02347657
How many people
510
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated

What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.

The limits of this study, and where it came from

Main limit. No limitation is recorded for this study.

How far it carries. This study is recorded from the curated record, not from the registry match.

Form and route. Oral

Interval reported. Not recorded in this summary

Written into the record, not signed off as a reviewed claim.

  • openFDA Drugs@FDA · a recorded source, not a stored snapshot
  • openFDA NDC Directory · a recorded source, not a stored snapshot
  • openFDA SPL label · a recorded source, not a stored snapshot
  • PubChem PUG-REST · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.

What is missing or unclearRead from sources, not yet reviewed

Were people like you studied?

RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.

Who was studied

People similar to this profile were included.

  • 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 [see Clinical Pharmacology (12.1) and Clinical Studies (14) ].

Who is missing from the studies

  • Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

What the label states about particular groups

  • On pediatric, the label states: “Safety of KALYDECO in patients aged 1 month and older was evaluated in a 96-week, open-label study (Trial 9) in 86 patients (38 rolled over from Trial 8, and 48 KALYDECO-naïve).”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

  • On older people, the label states: “Clinical trials of KALYDECO did not include sufficient numbers of patients 65 years of age and over to determine whether they respond differently from younger patients.”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

  • On people who are pregnant, the label states: “Risk Summary There are limited and incomplete human data from clinical trials and postmarketing reports on use of KALYDECO in pregnant women.”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

  • On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of ivacaftor in human milk, the effects on the breastfed infant, or the effects on milk production.”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

  • On people with reduced liver function, the label states: “Mild Hepatic Impairment (Child-Pugh Class A): No dosage adjustment is necessary for patients aged 6 months or older [see Clinical Pharmacology (12.3) ] .”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

  • On people with reduced kidney function, the label states: “KALYDECO has not been studied in patients with mild, moderate, or severe renal impairment or in patients with end-stage renal disease.”

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

Where the result stopped carrying

  • This is a scope explorer, not a diagnosis engine.
  • It shows who was studied so you can see whether people similar to you were included.

RNAWiki cannot tell whether a study fits you. It can show who was in it.

What it would be like to takeRead from sources, not yet reviewed

Why it might seem to do nothing

A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.

It was studied in different people

The people in the studies were not much like the reader.

On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

The evidence may simply be wrong

Small early studies often do not hold up.

On this record: RNAWiki has not yet published a reviewed conclusion for this use.

Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.

None of these is a reason to take more. Taking more is not a step this page ever suggests.

What it would be like to takeRead from sources, not yet reviewed

What taking it involves

The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.

How it is supplied

Prescription only

Quoted from a source

Where this came from

Copied from a source RNAWiki stored, with the source named.

Read from the recorded approval status.

No source is stored against this line.

Form and route

Oral

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

The form and route recorded on this substance.

No source is stored against this line.

Who oversees it

Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

Quoted from a source

Where this came from

Copied from a source RNAWiki stored, with the source named.

Suppression classes recorded: S6.

No source is stored against this line.

What is in the pack

Sold as tablet, granule, tablet, film coated, given by the oral route.

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

Recorded notes on how this is sold and what that means for what is in the pack.

No source is stored against this line.

Where it is registered

Regulatory records are listed in the technical disclosure at the foot of this page.

Counted from records

Where this came from

A count of rows RNAWiki holds. It describes our records, not your body.

Register entries are stored per jurisdiction and shown with their dates.

No source is stored against this line.

Why people stop

Not recorded.

Nothing recorded

Where this came from

RNAWiki holds nothing here and says so rather than guessing.

No record of why people stopped taking it is stored for this substance.

No source is stored against this line.

What it would be like to takeNothing found in the sources checked

What can go wrong

Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.

Nothing found in the sources checked

No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.

The sources listed were searched and held nothing. That is not the same as nothing existing.

Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.

Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.

What is missing or unclearRead from sources, not yet reviewed

Does the exact form matter?

Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.

The form that was studied

Oral

Source-linked record

Where this came from

A person wrote this into the record with the study named beside it. No reviewer has signed it off.

The form used in the studies cited on this page.

No source is stored against this line.

What is sold

Not recorded.

Nothing recorded

Where this came from

RNAWiki holds nothing here and says so rather than guessing.

Nothing further is recorded about which forms are sold.

No source is stored against this line.

What is recorded as being sold

  • 34 products list this as an active ingredient in the United States drug directory. 24 of them contain it and nothing else.

    FDA National Drug Code directory · 14501-0019 · read 2026-08-29

  • They are sold as granule, powder, tablet, coated and tablet, film coated, taken oral.

    FDA National Drug Code directory · 14501-0019 · read 2026-08-29

  • The regulator's established pharmacologic class for it is chloride channel activation potentiators [moa], cystic fibrosis transmembrane conductance regulator potentiator [epc] and cytochrome p450 2c9 inhibitors [moa].

    FDA National Drug Code directory · 14501-0019 · read 2026-08-29

  • 2 published labels name it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-29

  • Those labels are classed as human prescription drug.

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-29

  • Kalydeco is oral at 3 DOSAGE FORMS AND STRENGTHS Tablets: 150 mg, light blue, film-coated, oblong-shaped tablets, with the characters "V 150" on one side and plain on the other., recorded as fda label in effect 2026-03-23 in the United States.

    US prescribing information · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · read 2026-08-30

Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.

What it would be like to takeRead from sources, not yet reviewed

What you could measure

This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.

Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.

Questions worth asking

  • Which of the trials of Ivacaftor studied people like me?
  • What was measured, and for how long?
  • Was the result a laboratory value or a health outcome?
  • What would we watch for, and when would we stop?

Tracking can show whether something changed for you. It cannot show what caused it.

RNAWiki records evidence. It does not say whether this substance is right for you.

What is missing or unclearRead from sources, not yet reviewed

What nobody knows yet

Open questions, each with why it is open and what would close it.

Missing populations

Which groups were under-represented in the studies has not been recorded.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Missing long-term data

No completed tested study window is recorded.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

No reviewed conclusion

RNAWiki has not yet published a reviewed conclusion for this use.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Missing interaction studies

No interaction was found in the registers checked. Not finding one is not the same as showing there is none.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Formulation uncertainty

Several salts, forms or products of Ivacaftor are recorded. Results from one form may not transfer to another.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

Mechanism not reviewed

No reviewed mechanism story exists for this substance.

Why it matters. Recorded by the evidence model as a gap on this record.

What would answer it

A study that measures it, and a reviewer to sign it off.

Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.

What happened in peopleRead from sources, not yet reviewed

Check any of this yourself

Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.

Where else this substance is registered

FDA substance identifier (UNII)
1Y740ILL1Z
RxNorm concept
1243046

Checks this page had to pass

  • Passed

    Identity resolved

    no open identity hold

  • Passed

    No unresolved merge across substance families

    no quarantine open

  • Passed

    Every public sentence names a source

    The opening statement carries the origin: Written into the record, not signed off.

  • Not passed

    Trial roles classified for highlighted evidence

    No registered study is classified as testing this substance.

  • Passed

    No internal keys in reader text

    enforced by the copy-contract test over the rendered page

  • Passed

    Safety mode resolved

    Suppression classes recorded: S6.

  • Passed

    Canonical metadata present

    slug and display name present

What is missing or unclearRead from sources, not yet reviewed

How this medicine reached us

Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.

What the approval register records

  • 9 approved applications cover products containing this substance. The earliest was NDA203188, approved 20120131 to VERTEX PHARMS.

    Drugs@FDA application register · NDA203188 · read 2026-08-29

  • Marketing status on the register: none (tentative approval) and prescription.

    Drugs@FDA application register · NDA203188 · read 2026-08-29

  • The earliest marketing start date recorded for a listed product is 20120131.

    FDA National Drug Code directory · 14501-0019 · read 2026-08-29

What is missing or unclearRead from sources, not yet reviewed

What to learn next

Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.

This order is fixed in code and does not count clicks or time on the page.

What is not here

9 questions this page could not answer

These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.

  • What was measured, goal by goal — found nothing in the sources checked.
  • How close this is to real life — found nothing in the sources checked.
  • The path through the body — found nothing in the sources checked.
  • Felt, measured, or meaningful — found nothing in the sources checked.
  • How long anything takes — found nothing in the sources checked.
  • What it may clash with — found nothing in the sources checked.
  • Other ways to the same goal — found nothing in the sources checked.
  • Claims that go past the evidence — found nothing in the sources checked.
  • What changed on this page — found nothing in the sources checked.

The record as stored

The full record, for auditing

Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.

Recorded evidence blocks (10)

On the Ivacaftor label: indicated for what?


"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 [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . If the patient's…": indications and usage on Ivacaftor's label. DailyMed label · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · 2026-03-23

122 registered trials of Ivacaftor — at which phases?


Registered studies posting no result
44 of 122

122 registered studies of Ivacaftor: 57 phase3, 28 phase2, 17 phase1, 10 na or unstated, 5 na, 5 phase4, 3 early phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01

107 with a PubMed record

Show the evidence
  • phase3
    57
  • phase2
    28
  • phase1
    17
  • na or unstated
    10
  • na
    5
  • phase4
    5
8 more recorded rows
  • early phase1
    3
  • completed
    99
  • terminated
    7
  • active not recruiting
    6
  • approved for marketing
    3
  • recruiting
    3
  • withdrawn
    3
  • unknown
    1

recorded 2026-09-01 · last checked 2026-09-04

10 of Ivacaftor's trials stopped: futility/efficacy, accrual/recruitment, other?


futility/efficacy (2), accrual/recruitment (3) and other (5): Ivacaftor's stop wording, clustered. CLINICALTRIALS_SNAPSHOT · 2026-09-01

"Following review of results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility."; 10 of 122 registered studies

Show the evidence

Trial

  • NCT00953706
    terminated; "Following review of results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility."
  • NCT02653027
    withdrawn; "Potential subjects were already on the combination therapy."
  • NCT02742519
    terminated; "Low enrollment."
  • NCT02858843
    terminated; "We were unable to enroll eligible subjects for the study."
  • NCT02971839
    terminated; "Decision by Sponsor."
  • NCT03251911
    withdrawn; "no participants enrolled"
4 further recorded trials
  • NCT03447262
    terminated; "At Sponsor's Discretion"
  • NCT03633526
    terminated; "Study discontinued after Part A due to Sponsor's discretion."
  • NCT03956589
    terminated; "Patient inclusion has stopped after interim analysis."
  • NCT04066751
    withdrawn; "Study never started due to change in availability of study drug"

recorded 2026-09-01 · last checked 2026-09-04

Human studies of Ivacaftor used Ivacaftor 25 mg/75 mg — over how long?


studies of Ivacaftor used the recorded amount. ClinicalTrials.gov · 2026-09-01

5 recorded entries; human; also "Ivacaftor 25 mg/75 mg", "Ivacaftor 75 mg/150 mg", "Ivacaftor 150 mg or 250 mg"

Show the evidence

human

  • NCT00457821
    Ivacaftor 25 mg/75 mg
  • NCT00457821
    Ivacaftor 75 mg/150 mg
  • NCT00457821
    Ivacaftor 150 mg or 250 mg
  • NCT03085485
    Ivacaftor 150 MG
  • NCT07022327
    Ivacaftor 150 MG (milligrams)

recorded 2026-09-01 · last checked 2026-09-04

Ivacaftor's half-life is 12 hours — which schedules were studied?


12 hours, the half-life Ivacaftor's label states: "Elimination The apparent terminal half-life was approximately 12 hours following a single dose." DailyMed label · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · 2026-03-23

tmax 4 hours.

Show the evidence
  • half life pharmacokinetics
    12 hours; Elimination The apparent terminal half-life was approximately 12 hours following a single dose.
  • tmax pharmacokinetics
    4 hours; After oral administration of a single 150 mg dose to healthy volunteers in a fed state, peak plasma concentrations (T max ) occurred at approximately 4 hours, and the mean (±SD) for AUC and C max were 10600 (5260) ng*hr/mL and 768 (233) ng/mL, respectively.
  • metabolism pharmacokinetics
    Metabolism Ivacaftor is extensively metabolized in humans.

recorded 2026-03-23 · last checked 2026-09-04

Which 21 trials of Ivacaftor posted no result?


Posted no result
21 of 21 completed trials
Registrations
NCT00966602, NCT01018368, NCT01060566, NCT01153542, NCT01208285 and NCT01216046, and 15 more
Completion dates
oldest 2009-12; newest 2022-10-03
Show the evidence

Trial

  • NCT00966602
    2009-12
  • NCT01018368
    2010-03
  • NCT01060566
    2010-04
  • NCT01153542
    2010-08
  • NCT01208285
    2010-12
  • NCT01216046
    2011-05
14 further recorded trials
  • NCT01899105
    2013-08
  • NCT02015507
    2014-02
  • NCT01910415
    2014-03
  • NCT02392702
    2015-09
  • NCT02599792
    2016-04
  • NCT03486236
    2016-09-14
  • NCT03029455
    2017-08
  • NCT03475381
    2018-12-31
  • NCT04623879
    2020-02-26
  • NCT02445053
    2020-10
  • NCT03795363
    2021-06-16
  • NCT03783286
    2021-12-30
  • NCT04126473
    2022-04-06
  • NCT03894657
    2022-05-12

At the median, Ivacaftor's trials enrolled 70 people — anything larger?


Median enrolment
70
Largest enrolment
1164
Registered trials counted
119

What do 4506 spontaneous reports say about Ivacaftor — and not say?


These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.

Ivacaftor appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 4506 reaction mentions were counted: infective pulmonary exacerbation of cystic fibrosis 1801; hospitalisation 797; infection 388; cough 307. FAERS via Open Targets · CHEMBL2010601 · 2026-06-24

Show the evidence
  • infective pulmonary exacerbation of cystic fibrosis
    1801
  • hospitalisation
    797
  • infection
    388
  • cough
    307
  • cystic fibrosis
    293
  • pulmonary function test decreased
    217
4 more recorded rows
  • chest discomfort
    213
  • influenza
    175
  • haemoptysis
    166
  • cystic fibrosis respiratory infection suppression
    149

recorded 2026-06-24 · last checked 2026-09-04

Which 10 reactions does Ivacaftor's label not list?


chest discomfort, cough and cystic fibrosis and 7 more reported for Ivacaftor, absent from its label. FAERS via Open Targets · CHEMBL2010601 · 2026-06-24

2 label terms; 10 reported and unlisted; 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25

Show the evidence
  • chest discomfort
    count not stated
  • cough
    count not stated
  • cystic fibrosis
    count not stated
  • cystic fibrosis respiratory infection suppression
    count not stated
  • haemoptysis
    count not stated
  • hospitalisation
    count not stated
4 more recorded rows
  • infection
    count not stated
  • infective pulmonary exacerbation of cystic fibrosis
    count not stated
  • influenza
    count not stated
  • pulmonary function test decreased
    count not stated

recorded 2026-06-24 · last checked 2026-09-04

Ivacaftor and CYP3A: shared by which compounds?


CYP3A appear in Ivacaftor's recorded interaction sentences, 8 in all. DailyMed label · 0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25 · 2026-03-23

CYP3A; 1 shared nodes; drug_interactions

Show the evidence

Interaction statement

  • drug_interactions
    Potential for other drugs to affect ivacaftor CYP3A inhibitors: Reduce KALYDECO dosage in patients aged 6 months and older when co-administered with strong CYP3A inhibitors (e.g., ketoconazole) or moderate CYP3A inhibitors (e.g., fluconazole).
  • drug_interactions
    KALYDECO is not recommended in patients aged 1 month to less than 6 months when co-administered with strong or moderate CYP3A inhibitors.
  • drug_interactions
    ( 2.4 , 7.1 ) 7.1 Inhibitors of CYP3A Ivacaftor is a sensitive CYP3A substrate.
  • drug_interactions
    Co-administration with ketoconazole, a strong CYP3A inhibitor, significantly increased ivacaftor exposure [measured as area under the curve (AUC)] by 8.5-fold.
  • drug_interactions
    Based on simulations of these results, a reduction of the KALYDECO dosage is recommended for patients aged 6 months and older taking concomitant strong CYP3A inhibitors, such as ketoconazole, itraconazole, posaconazole, voriconazole, telithromycin, and clarithromycin.
  • drug_interactions
    KALYDECO is not recommended for patients less than 6 months of age taking strong CYP3A inhibitors [see Dosage and Administration (2.4) and Clinical Pharmacology (12.3) ] .
2 more recorded rows
  • Interaction statement drug_interactions
    Co-administration with fluconazole, a moderate inhibitor of CYP3A, increased ivacaftor exposure by 3-fold.
  • Interaction statement drug_interactions
    Therefore, a reduction of the KALYDECO dosage is recommended for patients aged 6 months and older taking concomitant moderate CYP3A inhibitors, such as fluconazole and erythromycin.
  • CYP3A
    FINGOLIMOD LAURYL SULFATE, Darunavir Propylene Glycolate, Vincristine, Naldemedine, Pemetrexed, Eravacycline, Rasburicase, Repotrectinib

recorded 2026-03-23 · last checked 2026-09-04

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL2010601
PubChem CID
46897962
CAS number
1246213-23-9
RxCUI
1243046
InChIKey
PURKAOJPTOLRMP-UHFFFAOYSA-N
Trade name
Ivacaftor component of orkambi, Ivacaftor component of symkevi, Ivacaftor component of trikafta, Kalydeco, ORKAMBI COMPONENT IVACAFTOR, SYMKEVI COMPONENT IVACAFTOR, TRIKAFTA COMPONENT IVACAFTOR, Kaftrio, Orkambi, Symkevi
Development code
VX-770
Also called
eti, iva, lumacaftor-ivacaftor, symdeko, Ivacaftor [INN], Ivacaftor [MI], Ivacaftor [ORANGE BOOK], Ivacaftor [USAN], Ivacaftor [VANDF], Ivacaftor [WHO-DD]
Sources (6)

Sources

ChEMBL 37 — CC BY-SA 3.0 Unported · ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work

How these records are assembled · Which registers were checked

Index-quality checks
  • identity passed: no open identity hold
  • required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
  • public claims reviewed: 0 reviewed claim(s); drafts are never rendered
  • source coverage passed: 6 source rows
  • no critical contamination: no quarantine open
  • canonical metadata passed: slug and display name present
  • no raw internal fields: enforced by the copy-contract test over the rendered page

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