Vanzacaftor-Tezacaftor-Deutivacaftor: A Once-Daily CFTR Modulator for Cystic Fibrosis

Table of Contents

1. What Correctors and Potentiators Do
2. How It Differs From the Older Triple Combination
3. The US Approval and Who the Label Covers
4. The Labelled Dose and the Fat-Containing Meal
5. The Boxed Liver Warning and Other Label Warnings
6. Interactions With Other Medicines and Grapefruit
7. The SKYLINE Trials in People Aged 12 and Older
8. Flare-Ups and Quality of Life in Later Analyses
9. The RIDGELINE Study in Children Aged 6 to 11
10. The TIMBERLINE Study in Children Aged 2 to 5
11. Side Effects in the Trials and Since Approval
12. What Is Not Yet Known
13. Key Research Papers
14. Connections
15. Featured Videos

What Correctors and Potentiators Do

Cystic fibrosis is caused by faults (variants) in both copies of the gene for the cystic fibrosis transmembrane conductance regulator, a channel in the surface of cells that lets chloride, a part of salt, flow in and out. When the channel is missing or stuck shut, the mucus in the lungs, pancreas and gut becomes thick and sticky, and sweat becomes unusually salty. The most common fault, called F508del, makes a misfolded protein that the cell mostly destroys before it ever reaches the surface. The Günter Blobel page explains why a protein that cannot be delivered is the core problem.

The drugs in this family come in two kinds, and a 2025 systematic review describes them as "correctors and potentiators":

• Correctors help the faulty protein fold and travel to the cell surface. According to the US label, vanzacaftor and tezacaftor bind to different sites on the protein and have an additive effect, increasing the amount of protein delivered to the surface compared with either drug alone.
• Potentiators work on protein that has already reached the surface, holding the channel open for longer. The label describes deutivacaftor as the potentiator in this tablet: it raises the channel's "open probability (or gating)".

The label sums up the combined effect as more channel protein at the cell surface that also works better, measured in the laboratory as chloride flow and in patients as a lower salt level in sweat. In cell studies, the combination increased the share of mature, fully processed channel protein more than elexacaftor-tezacaftor-ivacaftor did, while the immediate effect on channel function was similar; this was seen in 62 gut-lining organoid lines grown from patients' cells.

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How It Differs From the Older Triple Combination

Before this drug, the main triple combination was elexacaftor-tezacaftor-ivacaftor, approved in the United States in 2019. Vanzacaftor-tezacaftor-deutivacaftor keeps one ingredient (tezacaftor) and changes the other two:

• Vanzacaftor replaces elexacaftor as the second corrector.
• Deutivacaftor replaces ivacaftor as the potentiator. Deutivacaftor is ivacaftor with some of its hydrogen atoms swapped for deuterium, a heavier form of hydrogen. The US label calls it "a deuterated isotopologue of ivacaftor". A 2017 laboratory and volunteer study of this compound, then coded CTP-656, found that the swap made it markedly more stable against breakdown in laboratory tests while keeping the same strength against the channel in cells. In six healthy volunteers its half-life was 15.9 hours, and the authors wrote that this "suggests that CTP-656 may be dosed once daily".
• Once a day instead of twice. In the trials, elexacaftor-tezacaftor-ivacaftor was taken as two tablets in the morning plus one ivacaftor tablet in the evening; vanzacaftor-tezacaftor-deutivacaftor was taken as two tablets in the morning only. A 2026 drug review in an Australian prescribing journal gives deutivacaftor's half-life as 19 hours against 12 hours for ivacaftor. The label lists an effective half-life of about 93 hours for vanzacaftor, 23 hours for tezacaftor and 19 hours for deutivacaftor.

The trial authors write that once-daily dosing "reduces treatment burden, potentially improving adherence"; the trials themselves did not measure adherence.

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The US Approval and Who the Label Covers

According to the Food and Drug Administration's approval records, the drug was approved on December 20, 2024 as a new molecular entity. The indication was revised in March 2026. The label version read for this page (effective August 2026) words the indication this way: the treatment of cystic fibrosis "in adult and pediatric patients 6 years of age and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data … or results in production of CFTR protein."

• Age. The label covers people aged 6 and older. It states that safety and effectiveness "in patients younger than 6 years of age have not been established."
• Variants. The label's Table 5 lists the variants judged responsive. Those backed by clinical data include F508del, G551D, A455E, D1152H and about 20 others; several hundred more are listed from laboratory testing in cells. The list is described as "non-exhaustive". One variant, N1303K, is listed as responsive only on testing in human airway cells.
• Diagnosis first. The label states that the drug is meant only for people with a clinical diagnosis of cystic fibrosis, and that having an eligible variant is not on its own the deciding factor. When a person's genotype is unknown, the label calls for a genetic test cleared by the Food and Drug Administration.
• Children aged 6 to 11. The label explains that effectiveness in this group was extrapolated from the trials in people aged 12 and older, supported by blood-level data, while safety came from the RIDGELINE study described below.

Laboratory work since approval suggests the list may keep growing. A 2026 study tested 417 variants in cells: about 76% passed the standard laboratory threshold for responding to vanzacaftor-tezacaftor with ivacaftor, including 50 variants that had been classed as not responding or borderline to elexacaftor-tezacaftor-ivacaftor. A 2026 review warned that these laboratory thresholds have limits and that "clinical outcomes remain the ultimate determinant of therapeutic benefit."

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The Labelled Dose and the Fat-Containing Meal

The drug comes as two strengths of purple tablet, both containing all three drugs. The label's dosing table is:

• Age 12 and older, any weight: two tablets of vanzacaftor 10 mg / tezacaftor 50 mg / deutivacaftor 125 mg once a day (a total of 20 mg / 100 mg / 250 mg).
• Age 6 to 11, 40 kg or more: the same two tablets once a day.
• Age 6 to 11, under 40 kg: three tablets of vanzacaftor 4 mg / tezacaftor 20 mg / deutivacaftor 50 mg once a day (a total of 12 mg / 60 mg / 150 mg).

The tablets are swallowed whole, once a day at about the same time, with fat-containing food. The label gives examples of such meals: foods prepared with butter or oils, or containing eggs, peanut butter, cheeses, nuts, whole milk or meats. Food makes a large difference: compared with fasting, a fat-containing meal raised the amount of vanzacaftor absorbed about four-fold (lower-fat meal) to six-fold (high-fat meal), and deutivacaftor three- to four-fold. Tezacaftor was not meaningfully affected.

For a missed dose, the label says that if 6 hours or less have passed, the dose is taken as soon as possible; if more than 6 hours have passed, it is skipped and the usual schedule resumes the next day.

Before the first dose, the label calls for liver blood tests (alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase and bilirubin), then monthly for the first 6 months, every 3 months for the next 12 months, and at least yearly after that. It also calls for eye examinations before and during treatment in children (see below).

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The Boxed Liver Warning and Other Label Warnings

The boxed warning: drug-induced liver injury and liver failure

The label carries the Food and Drug Administration's strongest warning, a boxed warning, titled "Drug-Induced Liver Injury and Liver Failure". It states that raised liver enzymes have been seen in people taking vanzacaftor-tezacaftor-deutivacaftor, and that serious and potentially fatal liver injury and liver failure, leading to transplantation and death, were reported with elexacaftor-tezacaftor-ivacaftor, which "contains the same or similar active ingredients". With that older drug, liver injury was reported from the first month of treatment up to 15 months after starting. The label lists signs of liver injury that call for stopping the drug for a time: large rises in liver enzymes (more than 5 times the upper limit of normal, or more than 3 times with bilirubin more than 2 times normal), yellowing of the skin or eyes, pain under the right ribs, nausea, vomiting, confusion or fluid in the belly.

On liver disease, the label states the drug is not to be used in severe liver impairment (Child-Pugh class C), and advises against it in moderate impairment (Child-Pugh class B) except where there is a clear medical need and the benefit outweighs the risk.

Other warnings

• Allergic reactions, including anaphylaxis, reported after marketing with drugs containing elexacaftor, tezacaftor or ivacaftor.
• People who stopped earlier modulators because of side effects: the label states there are no safety data for this group, because they were excluded from the trials.
• Raised pressure inside the skull (intracranial hypertension), listed among the September 2025 label changes, from reports with drugs containing the same or similar ingredients. The label lists headache, blurred or double vision and possible vision loss, and notes that people with high vitamin A levels may be at greater risk.
• Neuropsychiatric events, including suicidal thoughts and behaviour, listed among the March 2026 label changes. Anxiety, depression, suicidal thoughts and behaviour, and sleep problems have been reported after marketing in adults and children, with and without past mental-health symptoms, and "may occur within the first three months" of starting.
• Cataracts. Clouding of the lens that was not present from birth has been reported in children and teenagers taking drugs that contain ivacaftor. The label says a link to ivacaftor "cannot be excluded" and calls for eye examinations before and during treatment in children. In young rats, ivacaftor caused cataracts.
• Drug interactions through the liver enzyme system, covered in the next section.

Contraindications and pregnancy

The label lists no contraindications. It states there are no human data on use in pregnancy; in rats and rabbits, vanzacaftor and tezacaftor caused no developmental harm at exposures below doses that were toxic to the mothers, and deutivacaftor is judged on ivacaftor's animal data.

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Interactions With Other Medicines and Grapefruit

All three drugs are broken down mainly by the liver enzymes cytochrome P450 3A4 and 3A5, often written CYP3A (cytochrome P450 3A). Medicines that speed up or slow down this enzyme change how much drug reaches the blood:

• Strong or moderate cytochrome P450 3A inducers (the Australian review gives phenytoin as an example of a strong inducer) lowered the levels of all three drugs. The label advises against using them together because the combination may work less well.
• Strong or moderate cytochrome P450 3A inhibitors (the Australian review gives clarithromycin and ketoconazole as examples) raised the levels of all three drugs. The label cuts the dose. For people aged 12 and over, the labelled dose with a moderate inhibitor is one tablet every other day, and with a strong inhibitor one tablet once a week.
• Grapefruit. Grapefruit juice contains compounds that moderately block cytochrome P450 3A, and the label tells patients to leave out food and drink containing grapefruit during treatment. The site's grapefruit interactions page explains the mechanism.
• Effects on other drugs. Tezacaftor and deutivacaftor block P-glycoprotein, a pump that moves many drugs out of cells, so levels of drugs that depend on it may rise. Vanzacaftor and deutivacaftor block the breast cancer resistance protein pump in laboratory tests. Deutivacaftor blocks the enzyme cytochrome P450 2C9 in laboratory tests, and the label calls for more frequent blood-clotting tests in people taking warfarin.
• Hormonal contraceptives. The label expects no meaningful interaction but notes that a role for hormonal contraceptives in rash "cannot be excluded".

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The SKYLINE Trials in People Aged 12 and Older

Approval rested on two 52-week, double-blind trials published in 2025, called SKYLINE 102 and SKYLINE 103. Every participant first took elexacaftor-tezacaftor-ivacaftor for 4 weeks, then was randomly assigned either to stay on it or to switch to vanzacaftor-tezacaftor-deutivacaftor. Everyone took dummy tablets so that nobody knew which drug they had.

• SKYLINE 102 enrolled 398 people with one F508del copy and one "minimal function" variant that makes little or no working protein. Median age was 31.
• SKYLINE 103 enrolled 573 people with two F508del copies, or F508del with a gating or residual-function variant, or other variants that respond to elexacaftor-tezacaftor-ivacaftor. Median age was 33.1.

Lung function: the main measure

The main measure was the change in forced expiratory volume in one second (FEV1), the amount of air blown out in one second, as a percentage of the value expected for a healthy person of the same size, age and sex. The question was whether the new drug was "non-inferior", meaning no more than 3 percentage points worse. Because everyone started on elexacaftor-tezacaftor-ivacaftor, lung function was already improved at the start; the trials tested whether switching kept it there.

• SKYLINE 102: +0.5 percentage points with vanzacaftor-tezacaftor-deutivacaftor against +0.3 with elexacaftor-tezacaftor-ivacaftor through week 24, a difference of 0.2 points (95% confidence interval −0.7 to 1.1).
• SKYLINE 103: +0.2 against 0.0, a difference of 0.2 points (−0.5 to 0.9).

Both trials met the non-inferiority test. Neither showed better lung function than the older drug.

Sweat chloride: a direct sign of channel function

Sweat chloride measures how well the channel works. A level of 60 mmol per litre or higher points to cystic fibrosis; below 30 is the normal range. The full trial report gives:

• Average change. Sweat chloride fell 8.4 mmol/L more with vanzacaftor-tezacaftor-deutivacaftor than with elexacaftor-tezacaftor-ivacaftor in SKYLINE 102 (95% confidence interval −10.5 to −6.3), and 2.8 mmol/L more in SKYLINE 103 (−4.7 to −0.9). The label puts average starting levels, after the run-in, at 53.9 mmol/L in the first trial and 42.8 in the second.
• Below 60 mmol/L (both trials pooled, through week 24): 86% (399 of 465) on vanzacaftor-tezacaftor-deutivacaftor against 77% (367 of 479) on elexacaftor-tezacaftor-ivacaftor. Put the other way, 14% and 23% stayed at or above 60.
• Below 30 mmol/L, the normal range: 31% (142 of 465) against 23% (108 of 479). Put the other way, 69% and 77% did not reach the normal range.

The trial authors called the new combination superior on sweat chloride. Whether that lower salt level turns into fewer illnesses or longer life over many years has not been measured; the authors wrote that "additional long-term data are needed".

Earlier phase 2 trials

Before SKYLINE, two short phase 2 trials in adults tested the combination. In people with two F508del copies who had been taking tezacaftor-ivacaftor, switching to vanzacaftor (20 mg)-tezacaftor-deutivacaftor for 4 weeks raised lung function by 15.9 percentage points and lowered sweat chloride by 45.5 mmol/L (18 people), against −0.1 points and −2.6 mmol/L for those who stayed on tezacaftor-ivacaftor (10 people).

Pooled comparisons

A 2025 network meta-analysis of 13 studies ranked vanzacaftor-tezacaftor-deutivacaftor first and elexacaftor-tezacaftor-ivacaftor second among the modulators for people with at least one F508del copy, and found a sweat chloride difference of 8.59 mmol/L between the two. A second 2025 network analysis of 29 studies reached similar rankings but warned that its results need cautious reading because of limited data and the low quality of the existing evidence.

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Flare-Ups and Quality of Life in Later Analyses

Two later analyses of the SKYLINE data, written with the manufacturer, looked beyond the main measures. Both were done after the trials ended (post hoc), which makes them weaker evidence than the planned results.

• Lung flare-ups (pulmonary exacerbations). In SKYLINE 102, the estimated flare-up rate was 0.26 per person per year on vanzacaftor-tezacaftor-deutivacaftor against 0.33 on elexacaftor-tezacaftor-ivacaftor (rate ratio 0.79, 95% confidence interval 0.54 to 1.15, which includes no difference). Flare-ups needing hospital care or intravenous antibiotics affected 5.1% against 12.9%. Intravenous antibiotic use for any reason was lower (12.2% against 24.8%; rate ratio 0.53, 0.30 to 0.93). In SKYLINE 103 the rates were similar in both groups (0.25 against 0.22 flare-ups per year).
• Quality of life. On a cystic fibrosis questionnaire scored as a single "utility" number, people aged 14 and older scored 0.017 higher on vanzacaftor-tezacaftor-deutivacaftor than on elexacaftor-tezacaftor-ivacaftor, with the larger gap in those with one minimal-function variant. The authors labelled these P values "nominal", meaning they were not adjusted for testing many outcomes.

The site's page on Pseudomonas lung infections in cystic fibrosis covers the germs behind many flare-ups.

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The RIDGELINE Study in Children Aged 6 to 11

RIDGELINE gave vanzacaftor-tezacaftor-deutivacaftor to 78 children aged 6 to 11 (median age 9.3) at 33 sites in eight countries, for 24 weeks. There was no comparison group. All children had at least one variant that responds to elexacaftor-tezacaftor-ivacaftor and had been taking that drug, so their starting values reflect it. Children under 40 kg took vanzacaftor 12 mg, tezacaftor 60 mg and deutivacaftor 150 mg; heavier children took the adult dose.

• Lung function started in the normal range (99.7% of predicted) and did not change (0.0 points; 95% confidence interval −2.0 to 1.9).
• Sweat chloride started at an average of 40.4 mmol/L and fell by 8.6 mmol/L. The share below 60 mmol/L rose from 84.4% to 94.9%; the share below 30 mmol/L rose from 39.0% to 52.6%, so just under half were still above the normal range.
• Side effects: 96% of children had at least one adverse event, all mild or moderate. The commonest were cough (46%), fever (21%), headache (18%), lung flare-up (17%) and sore throat (17%). Six children (8%) had a serious event, and one child stopped because of cough and tiredness judged possibly related to the drug. One child developed a mild cataract at the back of the lens on day 170 that did not affect vision. In the label's summary of this study, liver enzymes rose above 3 times normal in 3.8% of children and above 5 times normal in 1.3%.

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The TIMBERLINE Study in Children Aged 2 to 5

TIMBERLINE, published online in September 2026, studied preschool children aged 2 to 5 with a variant that responds to elexacaftor-tezacaftor-ivacaftor. Like RIDGELINE, it had no comparison group, and the children had been on elexacaftor-tezacaftor-ivacaftor beforehand. In the main 24-week part, run at 30 sites in nine countries, 67 children took a weight-based dose: under 12 kg, vanzacaftor 8 mg, tezacaftor 32 mg and deutivacaftor 100 mg; 12 kg or more, vanzacaftor 12 mg, tezacaftor 48 mg and deutivacaftor 150 mg, once a day.

• Side effects: 64 of 67 children (96%) had at least one adverse event, mostly mild (52%) or moderate (39%). Two children (3%) had serious events, judged unrelated to the drug. No child stopped because of side effects, and all 67 finished the study.
• Sweat chloride fell by an average of 9.6 mmol/L, to 28.9 mmol/L. By week 24, 61 of 66 children (92%) were below 60 mmol/L, leaving 5 (8%) at or above it; 43 (65%) were below 30 mmol/L, leaving 35% above the normal range.

At the time of writing, the US label still covers only people aged 6 and older.

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Side Effects in the Trials and Since Approval

In the SKYLINE trials

Almost everyone had at least one adverse event: 96% in each group, mostly mild or moderate, and many were ordinary features of cystic fibrosis. The commonest overall were lung flare-ups (28% on vanzacaftor-tezacaftor-deutivacaftor against 32% on elexacaftor-tezacaftor-ivacaftor), cough (23% against 21%), COVID-19 (22% against 26%) and common colds (21% against 19%). Serious adverse events affected 14% and 16%. The label lists side effects that were at least 1 percentage point more common on the new drug:

• Cough 25% against 24%; common cold 21% against 19%; upper airway infection 21% against 20%
• Headache 16% against 13%; sore throat 14% against 12%; tiredness 11% against 9%
• Influenza 11% against 5%; sinus congestion 7% against 3%
• Rash 11% against 7.7% (13% against 7.9% in women and girls), mostly mild to moderate
• Raised liver enzymes (alanine aminotransferase 8% against 6%; aspartate aminotransferase 7% against 5%)

Liver enzymes. Peak liver-enzyme levels above 3 times normal were seen in 6% against 3.1%, above 5 times in 2.5% against 1.2%, and above 8 times in 1.3% against 0.2%. Stopping because of raised liver enzymes occurred in 1.5% against 0.6%. The trial authors point out that everyone had already taken elexacaftor-tezacaftor-ivacaftor before randomisation, so early liver and rash reactions to that drug had already happened before the comparison began. First liver-enzyme rises and first rashes were more common on the new drug in the first 3 months and first month respectively, and similar after that. Overall, 3.8% stopped because of side effects on the new drug and 3.7% on the old one. The label also reports large rises in the muscle enzyme creatine phosphokinase in 7.9% against 6.5%.

Reports since approval

• Mood and mental health. At one adult centre in England, 87 people were switched to vanzacaftor-tezacaftor-deutivacaftor; 4 (4.6%) developed new or worse psychiatric symptoms on the standard dose, including suicidal thoughts. Three switched back to elexacaftor-tezacaftor-ivacaftor and their symptoms cleared within days. A 2026 analysis by authors including the manufacturer's scientists, covering more than 250,000 patient-years with modulators, did not find a raised risk of neuropsychiatric adverse events and found no off-target effects in laboratory or animal tests.
• A US case series. Of 38 adults switched at one centre in 2025, six had possible drug-related problems: raised liver enzymes, worse breathing symptoms, rashes, mood symptoms, stomach symptoms and high blood pressure. One was hospitalised with a severe rash, low platelets and raised liver enzymes. All problems cleared within days of switching back.

Reports of single centres cannot show how often a side effect happens across all users or prove that the drug caused it.

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What Is Not Yet Known

• Long-term outcomes. The longest controlled data run 52 weeks. No study yet shows whether the lower sweat chloride translates into slower lung decline, fewer hospital stays or longer life compared with elexacaftor-tezacaftor-ivacaftor. Survival projections on the cystic fibrosis page come from a computer model of the older drug, not this one.
• People new to modulators. In the SKYLINE, RIDGELINE and TIMBERLINE studies, nearly everyone had already taken elexacaftor-tezacaftor-ivacaftor, and the label notes that the adverse-reaction data come from people "who were able to tolerate" it. How the drug behaves in people starting a modulator for the first time is less well studied.
• People who could not tolerate earlier modulators. They were excluded, and the label states there are no safety data for them.
• Young children. TIMBERLINE was a single-group study of 67 children over 24 weeks. A 2026 Australian review noted that for ages 6 to 11, effectiveness "will need to be confirmed".
• Rare variants. Much of the eligible-variant list rests on cell tests rather than on people taking the drug.
• Pregnancy and breastfeeding. There are no human data.
• Who is left out. A 2026 review estimated that 10% to 15% of people with cystic fibrosis worldwide either do not qualify for or do not tolerate current modulators.
• Cost. None of the sources read for this page gave a price.

Every trial described on this page was funded by the manufacturer, and several authors are its employees.

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Key Research Papers

1. Keating C, Yonker LM, Vermeulen F, et al. Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. Lancet Respir Med. 2025;13(3):256-271. PubMed PMID: 39756424
2. Uluer AZ, MacGregor G, Azevedo P, et al. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023;11(6):550-562. PubMed PMID: 36842446
3. Hoppe JE, Kasi AS, Pittman JE, et al. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025;13(3):244-255. PubMed PMID: 39756425
4. Rayment JH, Davies G, Gaffin JM, et al. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 2-5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study. Lancet Respir Med. 2026 (online ahead of print). PubMed PMID: 42710511
5. Horsley A, Arteaga-Solis E, Keating C, et al. Decreased pulmonary exacerbations and lower intravenous antibiotic usage following vanzacaftor/tezacaftor/deutivacaftor treatment in people with cystic fibrosis with F508del-minimal function genotypes. Eur Respir J. 2026;67(6). PubMed PMID: 42025306
6. Wainwright CE, Thorat T, Conner S, et al. Improvements in health-related quality of life in people with cystic fibrosis ≥6 years of age treated with vanzacaftor/tezacaftor/deutivacaftor. J Cyst Fibros. 2026;25(2):284-289. PubMed PMID: 41350125
7. Harbeson SL, Morgan AJ, Liu JF, et al. Altering Metabolic Profiles of Drugs by Precision Deuteration 2: Discovery of a Deuterated Analog of Ivacaftor with Differentiated Pharmacokinetics for Clinical Development. J Pharmacol Exp Ther. 2017;362(2):359-367. PubMed PMID: 28611092
8. [No authors listed]. Vanzacaftor, tezacaftor and deutivacaftor fixed-dose combination for cystic fibrosis. Aust Prescr. 2026;49(5):204-206. PubMed PMID: 42936244
9. Iftikhar IH, Rao ST, Nadama R, et al. Comparative Efficacy of CFTR Modulators: A Network Meta-analysis. Lung. 2025;203(1):49. PubMed PMID: 40102290
10. Safeer V S M, Behl S, Vaidya PC, et al. Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysis. EClinicalMedicine. 2025;90:103655. PubMed PMID: 41377908
11. Kroes S, Zaidi L, Sonneveld-van Kooten HN, et al. A functional comparison of vanzacaftor/tezacaftor/deutivacaftor and elexacaftor/tezacaftor/ivacaftor in patient-derived intestinal organoids with rare CFTR variants. J Cyst Fibros. 2026;25(5):786-795. PubMed PMID: 42303492
12. Bihler H, Sivachenko A, Bhatt P, et al. In Vitro Responsiveness of 417 CFTR Variants to Vanzacaftor/Tezacaftor/Ivacaftor. J Cyst Fibros. 2026 (online ahead of print). PubMed PMID: 42728175
13. Rodriguez Mier N, Callebaut I, Proesmans M, et al. Rethinking CFTR variant responsiveness: Differential responses to vanzacaftor and elexacaftor. J Cyst Fibros. 2026;25(2):314-318. PubMed PMID: 41478784
14. Sawant AP, Shaw N, Clifton IJ, et al. Neuropsychiatric adverse effects following switch to vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: early experience from a regional centre. J Cyst Fibros. 2026 (online ahead of print). PubMed PMID: 42763277
15. Saag J, O'Bryant S, Amos K, et al. Adverse effects of vanzacaftor-tezacaftor-deutivacaftor in a cystic fibrosis population: A case series. Respir Med Case Rep. 2026;63:102474. PubMed PMID: 42633132
16. Van Goor F, Francioli L, Sosnay PR, et al. Evaluation of potential relationship between CFTR modulators, CFTR and neuropsychiatric symptoms in CF. J Cyst Fibros. 2026 (online ahead of print). PubMed PMID: 42946579
17. Fajac I, Jain R, Mall MA, et al. The clinical, psychosocial, and economic burden of cystic fibrosis lung disease in the era of CFTR modulator therapy. Ann Am Thorac Soc. 2026;23(1):17-29. PubMed PMID: 40961131

US prescribing information and approval history: DailyMed label (set ID 7e635909-c6fd-4f0d-ae77-cdff03653a20); Drugs@FDA, NDA 218730.

PubMed Topic Searches

1. PubMed: vanzacaftor
2. PubMed: deutivacaftor
3. PubMed: CFTR modulator liver injury
4. PubMed: CFTR modulator neuropsychiatric effects

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Connections

• Drugs — every drug page on the site
• Cystic Fibrosis — the disease this drug treats
• Cystic Fibrosis: History and Discovery — from the 1938 description to the first modulators
• Günter Blobel — why the F508del protein never arrives, and why correctors help
• Cystic Fibrosis Lung Infections — the infections behind lung flare-ups
• Bronchiectasis — the airway damage cystic fibrosis causes
• Grapefruit and Drug Interactions — why the label excludes grapefruit
• Cataracts — the lens clouding named in the label
• Pulmonology — all lung conditions on the site

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