Drugs / elexacaftor
elexacaftor
also known as
ELX · VX-445 · 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)- · Elexacaftor component of trikafta · elexacaftor-tezacaftor-ivacaftor · VX-445/TEZ/IVA
Small molecule targets CFTR
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed cystic fibrosis 5 milestones | |||||
| Filed | EU (EMA) | TRIKAFTA/KAFTRIO | cystic fibrosis | 2021-06-09 | “Vertex has submitted applications for use of TRIKAFTA/KAFTRIO in children ages 6 through 11 years to the European Medicines Agency (EMA)” vrtx.com ↗ |
| Filed | US (FDA) | TRIKAFTA | cystic fibrosis | 2021-01-26 | “FDA has accepted its supplemental New Drug Application (sNDA) to expand the use of TRIKAFTA®” vrtx.com ↗ |
| Filed | EU (EMA) | KAFTRIO | cystic fibrosis | 2020-09-14 | “EMA has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of KAFTRIO®*” vrtx.com ↗ |
| Filed | US (FDA) | TRIKAFTA | cystic fibrosis | 2020-09-01 | “FDA accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor).” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2019-10-31 | “Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen.” vrtx.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Kaftrio | — | 2020-08-21 | europa.eu ↗ |
| Approved | US (FDA) | TRIKAFTA (COPACKAGED) | — | 2019-10-21 | fda.gov ↗ |
Trials 24
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03227471 | Jan 2017 → Mar 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 21 trial · 1 missed | ||||||
| Phase 2 | NCT03506061 | Sep 2019 → Feb 2024 | cystic fibrosis | Emory University | Completed | Missed primary |
| Phase 315 trials · 4 met primary | ||||||
| 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 | Met primary |
| 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 | Met primary |
| 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 | NCT03525548 | Aug → Dec 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT03525444 | Jun 2018 → Apr 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 45 trials · 1 met primary | ||||||
| Phase 4 | NCT07809867 | Sep 2026 → Apr 2028 expected | cystic fibrosis | Gaslini Institute | Not yet recruiting | No outcome recorded |
| 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 | NCT04602468 | Sep 2020 → Jul 2029 expected | cystic fibrosis, cystic fibrosis-related liver disease | Royal College of Surgeons, Ireland | Active not recruiting | Met primary |
| 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 |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT05033080 Comparator | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
News releases announcing trial results or a regulatory action · 47
| Date | Issuer | Release |
|---|---|---|
| 2026-04-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces US FDA Approval for Label Extensions of ALYFTREK® and TRIKAFTA®, Expanding Availability of These Medicines to ~95% of All People With CF in the United States vrtx.com ↗
Additionally, the U.S. FDA has also expanded the indication statement for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages 2 and older. |
| 2025-04-07 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted regulatory approval for a label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis (CF). |
| 2025-02-28 | Vertex Pharmaceuticals Incorporated | Regulatory 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 | Regulatory Vertex Announces U.S. FDA Approval for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Include Additional Non-F508del TRIKAFTA-Responsive Variants vrtx.com ↗
In addition, safety information on liver injury and liver failure has been updated from warnings and precautions to a boxed warning. |
| 2023-11-24 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency (EMA) has validated a Type II variation application to the Marketing Authorization for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor. |
| 2023-11-23 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves KAFTRIO® in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5 vrtx.com ↗ |
| 2023-11-02 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present Data Highlighting Long-Term Benefits of TRIKAFTA® at the North American Cystic Fibrosis Conference vrtx.com ↗
This interim analysis through Week 144 of a 192-week open-label extension study of TRIKAFTA® in children 6 years of age and older with CF and at least one F508del allele showed sustained improvements in CFTR function and respiratory symptoms following the pivotal study. |
| 2023-09-15 | Vertex Pharmaceuticals Incorporated | Regulatory 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 | Regulatory 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 ↗
“With this approval, we now have the ability to treat young children with TRIKAFTA and can proactively address the underlying cause of their disease.” |
| 2022-11-03 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present New Data on its Portfolio of Cystic Fibrosis Medicines at the 2022 North American Cystic Fibrosis Conference vrtx.com ↗
Based on an interim analysis through Week 144 of the 192-week study, people receiving TRIKAFTA showed maintained improvements in lung function, respiratory symptoms and cystic fibrosis transmembrane conductance regulator (CFTR) function. |
| 2022-06-10 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference vrtx.com ↗ |
| 2022-04-20 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗ |
| 2022-01-11 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years vrtx.com ↗
We are delighted that KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now approved for these young patients in the European Union. |
| 2021-11-12 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗ |
| 2021-10-19 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference (NACFC) vrtx.com ↗
96-week interim results of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) study show no loss of pulmonary function in people with at least one F508del allele, a first for any CFTR modulator |
| 2021-08-26 | Vertex Pharmaceuticals Incorporated | Results 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 | Regulatory Vertex Announces National Reimbursement Agreement in France for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) and SYMKEVI® (tezacaftor/ivacaftor) for Eligible Cystic Fibrosis Patients vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced a national reimbursement agreement with the French Health Authorities for the cystic fibrosis (CF) medicines KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor and SYMKEVI® (tezacaftor/ivacaftor) in combination with ivacaftor. |
| 2021-06-18 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
“The approval of TRIKAFTA marks a significant milestone for Canadians with CF, their families and Vertex,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. |
| 2021-06-09 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years |
| 2021-06-09 | Vertex Pharmaceuticals Incorporated | Regulatory CORRECTING and REPLACING 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 ↗
With this approval approximately 1,500 children with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease for the first time |
| 2021-04-28 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis Patients 12 Years and Older With At Least One F508del Mutation in the CFTR gene vrtx.com ↗
“This indication extension is important as CF patients in Europe with gating and residual function mutations will have access to KAFTRIO for the first time,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. |
| 2021-03-26 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis With At Least One F508del Mutation vrtx.com ↗ |
| 2021-03-24 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Australian TGA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat People With Cystic Fibrosis Ages 12 Years and Older Who Have At Least One F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. |
| 2021-01-26 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces U.S. FDA Acceptance of Supplemental New Drug Application for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations vrtx.com ↗
The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. |
| 2020-12-21 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces FDA Approvals of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Use in People With CF With Certain Rare Mutations vrtx.com ↗
SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. |
| 2020-09-24 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators vrtx.com ↗
In the interim analysis, TRIKAFTA was generally well-tolerated, with no new safety concerns. |
| 2020-09-14 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Medicines Agency Type II Variation Marketing Authorization Application Validation for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination with ivacaftor in People with One Copy of the F508del Mutation vrtx.com ↗ |
| 2020-09-10 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Phase 3 Study for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children Ages 6-11 Years With Cystic Fibrosis to Support Submissions for Global Regulatory Approvals vrtx.com ↗
The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies. |
| 2020-09-01 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Accepts Vertex’s Supplemental New Drug Applications for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Additional CFTR Mutations vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor). |
| 2020-08-21 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis in People Ages 12 Years and Older vrtx.com ↗
Approval of the triple combination regimen also expands the number of treatment options available to people ages 12 years and older with CF who have two copies of the F508del mutation, the most common CF-causing mutation worldwide. |
| 2020-07-20 | Vertex Pharmaceuticals Incorporated | Results Positive Phase 3 Study Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older With Cystic Fibrosis Who Have One Copy of the F508del Mutation and One Gating or Residual Function Mutation vrtx.com ↗
The study met all secondary endpoints, including a statistically significant mean within-group reduction of 22.3 mmol/L from baseline in sweat chloride (p<0.0001). |
| 2020-06-26 | Vertex Pharmaceuticals Incorporated | Regulatory CHMP Grants Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With KALYDECO® (ivacaftor) in People Ages 12 and Older With Cystic Fibrosis With the Most Common Genotypes vrtx.com ↗ |
| 2019-10-31 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Medicines Agency Marketing Authorization Application Validation for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Triple Combination Treatment in Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. |
| 2019-10-21 | Vertex Pharmaceuticals Incorporated | Regulatory ADDING MULTIMEDIA FDA Approves TRIKAFTA™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older Who Have at Least One F508del Mutation vrtx.com ↗ |
| 2019-08-20 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. |
| 2019-07-22 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Submits New Drug Application to the U.S. FDA for Triple Combination Regimen of VX-445 (Elexacaftor), Tezacaftor and Ivacaftor in Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. |
| 2019-06-21 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to SYMDEKO. |
| 2019-05-30 | Vertex Pharmaceuticals Incorporated | Results Vertex Selects Triple Combination Regimen of VX-445, Tezacaftor and Ivacaftor to Submit for Global Regulatory Approvals in Cystic Fibrosis vrtx.com ↗
Vertex previously announced that both of these Phase 3 studies met their primary endpoints, and the company today announced the final results of these studies, including results for key secondary endpoints and safety data. |
| 2019-04-30 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO® (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age vrtx.com ↗ |
| 2019-03-06 | Vertex Pharmaceuticals Incorporated | Results Two Phase 3 Studies of the Triple Combination of VX-445, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis vrtx.com ↗ |
| 2018-11-27 | Vertex Pharmaceuticals Incorporated | Results Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis vrtx.com ↗ |
| 2018-10-24 | Vertex Pharmaceuticals Incorporated | Results Vertex Reports Third-Quarter 2018 Financial Results vrtx.com ↗
the study's Data Safety Monitoring Board (DSMB) recommended to stop the study early due to futility. |
| 2018-10-18 | Vertex Pharmaceuticals Incorporated | Results Vertex Data Presented at North American Cystic Fibrosis Conference (NACFC) Demonstrate Rapid Progress Toward Expanding and Enhancing Options for Treating the Underlying Cause of Cystic Fibrosis vrtx.com ↗
Phase 3 ARRIVAL data support treating the underlying cause of cystic fibrosis with KALYDECO® (ivacaftor) as early as six months of age |
| 2018-08-15 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO® (ivacaftor) as First and Only Medicine to Treat the Underlying Cause of CF in Children Ages 12 to <24 Months with Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO based on clinical and/or in vitro assay data. |
| 2018-08-07 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves ORKAMBI® (lumacaftor/ivacaftor) as First Medicine to Treat the Underlying Cause of Cystic Fibrosis for Children Ages 2-5 Years with Most Common Form of the Disease vrtx.com ↗
today announced the U.S. Food and Drug Administration (FDA) has approved ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years |
| 2018-06-28 | Vertex Pharmaceuticals Incorporated | Regulatory Health Canada Approves PrSYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada approved PrSYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older |
| 2018-02-01 | Vertex Pharmaceuticals Incorporated | Results Vertex Selects Two Next-Generation Correctors, VX-659 and VX-445, to Advance into Phase 3 Development as Part of Two Different Triple Combination Regimens for People with Cystic Fibrosis vrtx.com ↗
Initial data from the ongoing VX-659 and VX-445 studies were announced today and are below. |
All press releases naming this drug 53 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 30 sources stand behind the page.
| Field | Value | Cited 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 ↗21“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 ↗ |
| 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 ↗ |
| 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 |
| 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 |