Drugs / tezacaftor

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Symkevi 2018-10-31 europa.eu
Approved US (FDA) SYMDEKO (COPACKAGED) 2018-02-12 fda.gov

Trials 40 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 13 trials
Phase 1 NCT06299709 Mar → May 2024 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 1 NCT06154447 Dec 2023 → Jul 2026 overdue cystic fibrosis Vertex Pharmaceuticals Incorporated Active not recruiting No outcome recorded
Phase 1 NCT02015507 Jan → Feb 2014 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 25 trials
Phase 2 NCT03506061 Sep 2019 → Feb 2024 cystic fibrosis Emory University Completed No outcome recorded
Phase 2 NCT02730208 Sep 2016 → Jul 2018 cystic fibrosis Vertex Pharmaceuticals Incorporated 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 NCT01531673 Feb 2012 → Mar 2014 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 325 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 NCT05444257 Nov 2022 → Oct 2026 expected cystic fibrosis Vertex Pharmaceuticals Incorporated Active not recruiting No outcome recorded
Phase 3 NCT05422222 Jun 2022 → Jun 2030 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 NCT05033080 Sep 2021 → May 2023 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 43 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 not applicable2 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
Phase not stated2 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
Also used as a comparator or background therapy in 7 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT03029455 Background Nov 2016 → Aug 2017 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 22 trials
Phase 2 NCT04006873 Comparator Sep 2019 → Oct 2020 cystic fibrosis Nottingham University Hospitals NHS Trust Completed No outcome recorded
Phase 2 NCT02951195 Background Nov 2016 → Jan 2018 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 34 trials
Phase 3 NCT04105972 Comparator Oct 2019 → Jul 2020 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 3 NCT03525548 Comparator 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 Comparator May → Sep 2018 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded

Press releases naming this drug 62 releases

DateIssuerRelease
2025-07-14 Vertex Pharmaceuticals Incorporated Vertex Announces Broad Reimbursement Agreement With NHS England for ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor) an Innovative Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis vrtx.com
2025-03-07 Vertex Pharmaceuticals Incorporated Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis 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
2024-07-02 Vertex Pharmaceuticals Incorporated Vertex Announces FDA Acceptance of New Drug Application for Vanzacaftor/Tezacaftor/Deutivacaftor, a Next-In-Class Triple Combination Treatment for Cystic Fibrosis vrtx.com
2024-02-05 Vertex Pharmaceuticals Incorporated Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis 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
2023-04-26 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
2022-04-20 Vertex Pharmaceuticals Incorporated Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation vrtx.com
2022-03-26 Vertex Pharmaceuticals Incorporated Vertex Announces Reimbursement Agreement in Australia for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for Patients With Cystic Fibrosis Ages 12 Years and Older With at Least One F508del Mutation in the CFTR Gene vrtx.com
2022-01-11 Vertex Pharmaceuticals Incorporated European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years vrtx.com
2021-11-19 Vertex Pharmaceuticals Incorporated Vertex Announces Reimbursement Agreement in Spain for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis 12 Years and Older With At Least One F508del Mutation in the CFTR Gene vrtx.com
2021-11-12 Vertex Pharmaceuticals Incorporated Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor in Children With Cystic Fibrosis Ages 6 Through 11 vrtx.com

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 51 sources stand behind the page.

FieldValueCited text
Known as Tezacaftor “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
41

“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

“BACKGROUND: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor-tezacaftor-deutivacaftor has been shown to be a safe and effective, once-daily...” PMID 39756425 Jan 2025

“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 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

“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

“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

“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

“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

“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 purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.” NCT05444257

“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

“vanzacaftor (20 mg once daily), tezacaftor (100 mg once daily), and deutivacaftor (250 mg once daily) as two fixed-dose combination tablets in the morning” PMID 39756424 Jan 2025

“elexacaftor-tezacaftor-ivacaftor/deutivacaftor; VX-659-tezacaftor-ivacaftor/deutivacaftor; VX-440-tezacaftor-ivacaftor; VX-152-tezacaftor-ivacaftor” PMID 37983082 Nov 2023

“Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del” NCT05331183

“The mean increase in CFQ scores with twice-daily tezacaftor (100 mg) and ivacaftor (150 mg) was approximately five points” PMID 33331662 Dec 2020

“Trikafta (Elexacaftor \[ELX\], Tezacaftor \[TEZ\], Ivacaftor\[IVA\]) is contraindicated” NCT04840862

“Standard of care: Lumacaftor with Ivacaftor or Tezacaftor combined with Ivacaftor.” NCT04415268

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.” PMID 29099333 Nov 2017

“Participants received oral tezacaftor 100 mg once daily” PMID 33581080 Feb 2021

“elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)” NCT05111145

“Vanzacaftor/tezacaftor/deutivacaftor” NCT06299709

“elexacaftor/tezacaftor/ivacaftor” NCT05274269

NCT02508207

NCT02730208

NCT06154447

NCT03150719

NCT04006873

NCT03278314

NCT03029455

NCT07148739

Known as TEZ “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
20

“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

“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

“Trikafta (Elexacaftor \[ELX\], Tezacaftor \[TEZ\], Ivacaftor\[IVA\]) is contraindicated” NCT04840862

“administered TEZ 100 milligram (mg)/IVA 150 mg fixed-dose tablet” NCT02565914

“VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA)” NCT02508207

“elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)” NCT05111145

Known as Tezacaftor component of symkevi ChEMBL registry synonym — accepted as the source's own label CHEMBL3544914
Known as Tezacaftor component of trikafta ChEMBL registry synonym — accepted as the source's own label CHEMBL3544914
Known as VX-661 “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
5

“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

NCT01531673

NCT02516410

NCT02015507

NCT02070744

Action Restore “This review evaluates single correctors (monotherapy) and any combination of correctors (most commonly lumacaftor, tezacaftor, elexacaftor, VX-659, VX-440 or VX-152) and a...” PMID 37983082 Nov 2023
Modality Other / unclassified “CFTR modulator (tezacaftor-ivacaftor,TEZ/IVA)” PMID 39139270 Mar 2024
Route Oral “VX-661 10 milligram (mg) tablet orally once daily (qd)” NCT01531673
Target CFTR “cystic fibrosis transmembrane conductance regulator (CFTR) modulators” PMID 29099344 Nov 2017