Regulatory milestones approvals, filings & regulatory actions · 13 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (11) | US (FDA) | KALYDECO | — | 2014-02-21 – 2025-05-22 | fda.gov ↗ |
| Approved | EU (EMA) | Kalydeco | — | 2012-07-23 | europa.eu ↗ |
| Approved | US (FDA) | KALYDECO | — | 2012-01-31 | fda.gov ↗ |
Trials 93 · started per year
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 02 trials | ||||||
| Phase 0 | NCT03439865 | May 2019 → Apr 2027 expected | chronic rhinosinusitis | University of Alabama at Birmingham | Recruiting | No outcome recorded |
| Phase 0 | NCT02135432 | May → Oct 2015 | chronic obstructive pulmonary disease | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 111 trials | ||||||
| Phase 1 | NCT02015507 | Jan → Feb 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01899105 | Jul → Aug 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01888393 | Jun → Nov 2013 | — | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01910415 | Jun 2013 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01768663 | Jan → Sep 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01216046 | Oct 2010 → May 2011 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01208285 | Sep → Dec 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01153542 | Jun → Aug 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01060566 | Feb → Mar 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01018368 | Nov 2009 → Mar 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT00966602 | Sep → Dec 2009 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 217 trials | ||||||
| Phase 2 | NCT04581408 | Jun 2021 → Apr 2025 | long QT syndrome | Istituto Auxologico Italiano | Completed | No outcome recorded |
| Phase 2 | NCT04135495 | Nov 2019 → Oct 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04126473 | Nov 2019 → Apr 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT03506061 | Sep 2019 → Feb 2024 | cystic fibrosis | Emory University | Completed | No outcome recorded |
| Phase 2 | NCT03625466 | Aug 2018 → Oct 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT03061331 | Jan → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT02730208 | Sep 2016 → Jul 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT02871778 | Aug 2016 → Nov 2018 | primary ciliary dyskinesia | Parion Sciences | Completed | No outcome recorded |
| Phase 2 | NCT02508207 | Feb 2016 → Jun 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT02070744 | Mar 2014 → May 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01685801 | Sep 2012 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01531673 | Feb 2012 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01262352 | Jan → Nov 2011 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01161537 | Oct 2010 → Feb 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01225211 | Oct 2010 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT00953706 | Sep 2009 → Jul 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 2 | NCT00457821 | May 2007 → Aug 2008 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 346 trials | ||||||
| Phase 3 | NCT06460506 | Nov 2024 → Sep 2027 expected | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05331183 | Nov 2022 → Apr 2027 expected | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05274269 | May 2022 → Jul 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT05153317 | Jan 2022 → Jan 2026 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT05111145 | Jan → Dec 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04969224 | Oct 2021 → Jul 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04545515 | Jan 2021 → Mar 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04362761 | May 2020 → Dec 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04235140 | Feb 2020 → Aug 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04058366 | Dec 2019 → Dec 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04105972 | Oct 2019 → Jul 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04043806 | Aug 2019 → Dec 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03525574 | Oct 2018 → Jan 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03691779 | Oct 2018 → Aug 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03633526 | Aug 2018 → Jan 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 3 | NCT03559062 | May → Dec 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03537651 | Apr 2018 → Oct 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03447249 | Mar 2018 → Feb 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03277196 | Aug 2017 → Oct 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03068312 | Jul 2017 → Dec 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03150719 | May 2017 → Aug 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03125395 | May 2017 → Jul 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02934698 | Nov 2016 → May 2018 | cystic fibrosis | Medical University of South Carolina | Completed | No outcome recorded |
| Phase 3 | NCT02725567 | Jun 2016 → Jun 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02797132 | May 2016 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02742519 | May 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 3 | NCT02516410 | Aug 2015 → Jun 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02565914 | Aug 2015 → May 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02544451 | Aug 2015 → Aug 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02514473 | Jul 2015 → Sep 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02412111 | Jun 2015 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02392234 | Mar 2015 → Feb 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02390219 | Mar 2015 → Oct 2016 | cystic fibrosis, lung disorder | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02347657 | Jan 2015 → Jan 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01946412 | Dec 2013 → Dec 2015 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01931839 | Oct 2013 → Apr 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01897233 | Jul 2013 → Oct 2015 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01807923 | May 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01807949 | Apr 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01707290 | Feb 2013 → Apr 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01705145 | Jan 2013 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01614470 | Jul 2012 → Oct 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01614457 | Jul 2012 → Oct 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01117012 | Jul 2010 → May 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT00909727 | Aug 2009 → Nov 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT00909532 | Jun 2009 → Jul 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 48 trials | ||||||
| Phase 4 | NCT07148739 | Jun 2025 → Dec 2030 expected | cystic fibrosis | Arkansas Children's Hospital Research Institute | Recruiting | No outcome recorded |
| Phase 4 | NCT05743946 | Apr 2023 → Dec 2025 | bronchiectasis | Emory University | Completed | No outcome recorded |
| Phase 4 | NCT04840862 | May 2022 → May 2024 | — | University of Southern California | Completed | No outcome recorded |
| Phase 4 | NCT03251911 | Jan 2020 → Feb 2022 | chronic bronchitis | University of Alabama at Birmingham | Withdrawn | No outcome recorded |
| Phase 4 | NCT03624101 | Dec 2018 → Oct 2019 | cystic fibrosis | University of Alabama at Birmingham | Terminated | No outcome recorded |
| Phase 4 | NCT03956589 | Oct 2018 → Apr 2020 | cystic fibrosis | University Hospital, Antwerp | Terminated | No outcome recorded |
| Phase 4 | NCT03256799 | Mar → Jul 2017 | cystic fibrosis | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 4 | NCT02875366 | Sep 2016 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase not applicable6 trials | ||||||
| — | NCT04580368 | May 2021 → Dec 2028 expected | cystic fibrosis | Children's Hospital Medical Center, Cincinnati | Recruiting | No outcome recorded |
| — | NCT04415268 | Jun 2020 → Jan 2021 | cystic fibrosis | Universidad Europea de Madrid | Withdrawn | No outcome recorded |
| — | NCT02653027 | Jan → Feb 2018 | cystic fibrosis, diabetes mellitus | Massachusetts General Hospital | Withdrawn | No outcome recorded |
| — | NCT02858843 | Aug 2016 → May 2018 | cystic fibrosis, diabetes mellitus | Massachusetts General Hospital | Terminated | No outcome recorded |
| — | NCT02310789 | Jul 2015 → Aug 2016 | cystic fibrosis | Richard Barry Moss | Completed | No outcome recorded |
| — | NCT01784419 | Oct 2013 → Dec 2015 | cystic fibrosis | University of California, San Francisco | Completed | No outcome recorded |
| Phase not stated3 trials | ||||||
| — | NCT04058210 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
| — | NCT03278314 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
| — | NCT01381289 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
Also used as a comparator or background therapy in 18 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT03029455 Background | Nov 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT02599792 Comparator | Nov 2015 → Apr 2016 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02392702 Comparator | Mar → Jun 2015 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 26 trials | ||||||
| Phase 2 | NCT06237335 Background | Feb 2024 → Aug 2026 overdue | cystic fibrosis | ReCode Therapeutics | Recruiting | No outcome recorded |
| Phase 2 | NCT04066751 Comparator | Dec 2021 → Oct 2024 | chronic obstructive pulmonary disease | University of Alabama at Birmingham | Withdrawn | No outcome recorded |
| Phase 2 | NCT04006873 Comparator | Sep 2019 → Oct 2020 | cystic fibrosis | Nottingham University Hospitals NHS Trust | Completed | No outcome recorded |
| Phase 2 | NCT03085485 Comparator | Mar 2017 → Sep 2022 | chronic bronchitis | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 2 | NCT02951195 Background | Nov 2016 → Jan 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT02709109 Background | Feb 2016 → Sep 2017 | cystic fibrosis | Parion Sciences | Completed | No outcome recorded |
| Phase 36 trials | ||||||
| Phase 3 | NCT05033080 Comparator | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04183790 Background | Feb 2020 → Feb 2024 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04058353 Background | Aug 2019 → Jun 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03525548 Background | Aug → Dec 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03447262 Background | Jul 2018 → Sep 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 3 | NCT03460990 Background | May → Sep 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 42 trials | ||||||
| Phase 4 | NCT02823470 Background | Jun 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 4 | NCT01937325 Comparator | Feb → Dec 2014 | cystic fibrosis | The Alfred | Unknown | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT03894657 Background | Dec 2019 → Nov 2021 | cystic fibrosis | Assistance Publique - Hôpitaux de Paris | Completed | No outcome recorded |
Press releases naming this drug 161 releases
| Date | Issuer | Release |
|---|---|---|
| 2025-04-07 | Vertex Pharmaceuticals Incorporated | European Commission Approves Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis vrtx.com ↗ |
| 2025-02-28 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinion for Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis to Include Rare Mutations vrtx.com ↗ |
| 2024-12-20 | Vertex Pharmaceuticals Incorporated | Vertex Announces U.S. FDA Approval for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Include Additional Non-F508del TRIKAFTA-Responsive Variants vrtx.com ↗ |
| 2024-04-26 | Vertex Pharmaceuticals Incorporated | Vertex Announces European Commission Approval for KALYDECO® to Treat Infants With Cystic Fibrosis Ages 1 Month and Older vrtx.com ↗ |
| 2024-02-23 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinion for KALYDECO® for the Treatment of Infants With Cystic Fibrosis Ages 1 Month and Older vrtx.com ↗ |
| 2023-11-24 | Vertex Pharmaceuticals Incorporated | Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO® in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations vrtx.com ↗ |
| 2023-11-23 | Vertex Pharmaceuticals Incorporated | European Commission Approves KAFTRIO® in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5 vrtx.com ↗ |
| 2023-09-15 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5 vrtx.com ↗ |
| 2023-07-05 | Vertex Pharmaceuticals Incorporated | European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old vrtx.com ↗ |
| 2023-05-03 | Vertex Pharmaceuticals Incorporated | Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older vrtx.com ↗ |
| 2023-04-27 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old vrtx.com ↗ |
| 2023-04-26 | Vertex Pharmaceuticals Incorporated | Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations vrtx.com ↗ |
| Show all 161 releases opens the full record — a long page | ||
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 116 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ivacaftor | “received weight-based and age-based doses of oral lumacaftor-ivacaftor: children weighing less than 14 kg and aged younger than 6 years at study 116 screening received...” PMID 33965000 ↗ May 202196“This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in...” NCT04058366 ↗ “This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects...” NCT04058353 ↗ “The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age...” NCT04058210 ↗ “Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in...” NCT03460990 ↗ “This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in...” NCT03633526 ↗ “This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in...” NCT02951195 ↗ “The CFTR modulating drugs that are currently FDA approved and will be tested in this study include ivacaftor and the combination drugs orkambi (ivacaftor/lumacaftor), symdeko...” NCT04580368 ↗ “This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis...” NCT03447262 ↗ “A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic...” NCT03559062 ↗ “This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous...” NCT03447249 ↗ “This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in...” NCT04105972 ↗ “Participants will take Trikafta which is a combination tablet comprised of 100 milligrams (mg) of elexacaftor, 50 mg of tezacaftor and 75 mg of ivacaftor (2 tablets taken in...” NCT03506061 ↗ “After a 4-week tezacaftor plus ivacaftor run-in period, participants were randomly assigned (1:1) to 4 weeks of elexacaftor 200 mg orally once daily plus tezacaftor 100 mg...” PMID 31679946 ↗ Oct 2019 “Rationale: Elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) was shown to be safe and efficacious in children 6 through 11 years of age with cystic fibrosis (CF) and at least...” PMID 40454869 ↗ Oct 2025 “The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF...” NCT05153317 ↗ “Participants will be given elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg (two pills once daily in the morning) and ivacaftor 150 mg (once daily in the evening), as the...” NCT05743946 ↗ “Over 120 weeks (initial study period and follow-up), systolic blood pressure rose by 5.1 mmHg and diastolic blood pressure by 4.1 mmHg with twice-daily 400 mg...” PMID 37983082 ↗ Nov 2023 “elexacaftor (200 mg once daily), tezacaftor (100 mg once daily), and ivacaftor (150 mg once every 12 h) as two fixed-dose combination tablets in the morning and one ivacaftor...” PMID 39756424 ↗ Jan 2025 “This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with...” NCT04043806 ↗ “the mean increase in CFQ scores with twice-daily tezacaftor (100 mg) and ivacaftor (150 mg) was approximately five points (95% CI 3.20 to 7.00; 504 participants;...” PMID 33331662 ↗ Dec 2020 “Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial.” PMID 39173175 ↗ Nov 2024 “Participants weighing less than (\<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h)” NCT03537651 ↗ “The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).” NCT06460506 ↗ “This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.” NCT04969224 ↗ “A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele.” PMID 33734030 ↗ Jun 2021 “Orkambi® is a combination of Ivacaftor (a CFTR channel potentiator) and Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR).” NCT03894657 ↗ “Infants received 25 mg or 50 mg ivacaftor every 12 hours on the basis of age and weight for 4 days in part A and 24 weeks in part B.” PMID 33023304 ↗ Mar 2021 “Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del” NCT05331183 ↗ “Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).” NCT02544451 ↗ “Trikafta (Elexacaftor \[ELX\], Tezacaftor \[TEZ\], Ivacaftor\[IVA\]) is contraindicated” NCT04840862 ↗ “Standard of care: Lumacaftor with Ivacaftor or Tezacaftor combined with Ivacaftor.” NCT04415268 ↗ “Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation.” PMID 33249003 ↗ Nov 2020 “Children received lumacaftor 100 mg and ivacaftor 125 mg” PMID 30686767 ↗ Jan 2019 “Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function” NCT03956589 ↗ “ivacaftor 150 mg once every 12 h for up to 96 weeks” PMID 33581080 ↗ Feb 2021 “elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)” NCT05111145 ↗ “lumacaftor/ivacaftor” NCT02823470 ↗ “Lumacaftor/Ivacaftor” NCT02875366 ↗ “ivacaftor” NCT04362761 ↗ “ivacaftor” NCT05274269 ↗ |
| Known as | IVA | “This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in...” NCT04058366 ↗26“This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects...” NCT04058353 ↗ “The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age...” NCT04058210 ↗ “Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in...” NCT03460990 ↗ “This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in...” NCT03633526 ↗ “This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in...” NCT02951195 ↗ “This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis...” NCT03447262 ↗ “This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous...” NCT03447249 ↗ “This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in...” NCT04105972 ↗ “Rationale: Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis and at least one F508del-CFTR...” PMID 33734030 ↗ Jun 2021 “The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF...” NCT05153317 ↗ “This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with...” NCT04043806 ↗ “Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks.” NCT04362761 ↗ “Participants weighing less than (\<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h)” NCT03537651 ↗ “The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).” NCT06460506 ↗ “This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.” NCT04969224 ↗ “Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning” NCT01931839 ↗ “This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF)” NCT03525548 ↗ “Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).” NCT02544451 ↗ “Trikafta (Elexacaftor \[ELX\], Tezacaftor \[TEZ\], Ivacaftor\[IVA\]) is contraindicated” NCT04840862 ↗ “it is combined with ivacaftor (IVA), an enhancer of the CFTR protein function.” NCT04581408 ↗ “VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy” NCT02392234 ↗ “VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA)” NCT02508207 ↗ “elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)” NCT05111145 ↗ “IVA (ivacaftor) 75 mg every 12 hours” NCT04183790 ↗ “Ivacaftor (IVA) 150 mg tablet” NCT02070744 ↗ |
| Known as | IVA, VX-770 | “IVA, VX-770” NCT02392234 ↗ |
| Known as | Kalydeco | “This is a prospective randomized double-blinded placebo-controlled crossover study of the short-term effects of ivacaftor (aka VX-770 or Kalydeco) on sweat chloride...” NCT01784419 ↗4“The new drug, Ivacaftor (KALYDECO), is a drug that has recently been approved by the U.S. Food and Drug Administration (FDA) for patients with a lung disease called Cystic...” NCT03251911 ↗ “All participants in group 7 who received placebo matched to VX-661 tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days.” NCT01531673 ↗ |
| Known as | Orkambi | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02709109 ↗ |
| Known as | Symdeko | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03624101 ↗ |
| Known as | VX-770 | “This is a prospective randomized double-blinded placebo-controlled crossover study of the short-term effects of ivacaftor (aka VX-770 or Kalydeco) on sweat chloride...” NCT01784419 ↗16“Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the...” PMID 29099344 ↗ Nov 2017 “The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have...” NCT01262352 ↗ “Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning” NCT01931839 ↗ “lumacaftor (VX-809), a CFTR corrector, in combination with ivacaftor (VX-770), a CFTR potentiator” PMID 25981758 ↗ May 2015 “Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator” PMID 23590265 ↗ Jun 2013 “VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy” NCT02392234 ↗ “Ivacaftor (VX-770)” NCT03251911 ↗ |
| Action | Activate | “VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro.” PMID 21083385 ↗ Nov 2010 |
| Modality | Small molecule | “orally bioavailable small molecule” NCT01381289 ↗ |
| Route | Oral | “receive oral VX-770 every 12 hours” PMID 21083385 ↗ Nov 2010 |
| Target | CFTR | “Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein.” NCT00457821 ↗ |