Drugs / elexacaftor

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Kaftrio 2020-08-21 europa.eu
Approved US (FDA) TRIKAFTA (COPACKAGED) 2019-10-21 fda.gov

Trials 22 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/21 trial
Phase 1/2 NCT03227471 Jan 2017 → Mar 2018 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded
Phase 21 trial
Phase 2 NCT03506061 Sep 2019 → Feb 2024 cystic fibrosis Emory University Completed No outcome recorded
Phase 315 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 NCT04362761 May 2020 → Dec 2022 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 NCT04058353 Aug 2019 → Jun 2020 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 applicable1 trial
NCT04580368 May 2021 → Dec 2028 expected cystic fibrosis Children's Hospital Medical Center, Cincinnati Recruiting No outcome recorded
Phase not stated1 trial
NCT04058210 cystic fibrosis Vertex Pharmaceuticals Incorporated Approved for marketing No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 3 NCT05033080 Comparator Sep 2021 → May 2023 cystic fibrosis Vertex Pharmaceuticals Incorporated Completed No outcome recorded

Press releases naming this drug 35 releases

DateIssuerRelease
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
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
2021-08-26 Vertex Pharmaceuticals Incorporated Vertex Announces Publication in The New England Journal of Medicine of Phase 3 Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People With Cystic Fibrosis vrtx.com
2021-06-28 Vertex Pharmaceuticals Incorporated Vertex Announces National Reimbursement Agreement in France for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) and SYMKEVI® (tezacaftor/ivacaftor) for Eligible Cystic Fibrosis Patients vrtx.com
2021-06-18 Vertex Pharmaceuticals Incorporated Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older Who Have at Least One F508del Mutation vrtx.com
2021-06-09 Vertex Pharmaceuticals Incorporated Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations vrtx.com

Evidence & citations 11 cited values

Every value below carries the sentence it was read from. 28 sources stand behind the page.

FieldValueCited text
Known as elexacaftor “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
19

“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

“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 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

“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

“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

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 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

“The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes” PMID 31679946 Oct 2019

“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

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

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

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

elexacaftor/tezacaftor/ivacaftor” NCT05274269

NCT07148739

Known as 3-PYRIDINECARBOXAMIDE, N-((1,3-DIMETHYL-1H-PYRAZOL-4-YL)SULFONYL)-6-(3-(3,3,3-TRIFLUORO-2,2-DIMETHYLPROPOXY)-1H-PYRAZOL-1-YL)-2-((4S)-2,2,4-TRIMETHYL-1-PYRROLIDINYL)- ChEMBL registry synonym — accepted as the source's own label CHEMBL4298128
Known as Elexacaftor component of trikafta ChEMBL registry synonym — accepted as the source's own label CHEMBL4298128
Known as elexacaftor-tezacaftor-ivacaftor “Patients were randomly assigned to receive elexacaftor-tezacaftor-ivacaftor or placebo for 24 weeks.” PMID 31697873 Oct 2019
Known as ELX “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
12

“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

“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

“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

“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

“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

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

“Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks” NCT05033080

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

Known as VX-445 “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
5

“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

“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

“The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes” PMID 31679946 Oct 2019

NCT03227471

Known as VX-445/TEZ/IVA ClinicalTrials.gov intervention name — accepted as the source's own label NCT03525444
Action Restore “VX-445 is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function” PMID 30334692 Oct 2018
Modality Small molecule “Elexacaftor-tezacaftor-ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen” PMID 34437784 Aug 2021
Route Oral “VX-445 tablet for oral administration.” NCT03227471
Target CFTR “VX-445 is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function” PMID 30334692 Oct 2018