Drugs / tezacaftor
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed cystic fibrosis 5 milestones | |||||
| Filed | US (FDA) | — | cystic fibrosis | 2024-07-02 | “today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor...” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2019-10-31 | “today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor...” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2019-07-22 | “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...” vrtx.com ↗ “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.” vrtx.com ↗ “In late 2018, Vertex submitted an sNDA to the U.S. Food and Drug Administration (FDA) for tezacaftor/ivacaftor” vrtx.com ↗ “sNDAs were submitted to the FDA for ivacaftor in infants ages 6 to <12 months and for tezacaftor/ivacaftor” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2019-04-30 | “Supplemental New Drug Application (sNDA) submitted in the U.S. for tezacaftor/ivacaftor in children ages 6 to 11 years old.” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2018-02-12 | “The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the tezacaftor/ivacaftor combination.” vrtx.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Symkevi | — | 2018-10-31 | europa.eu ↗ |
| Approved | US (FDA) | SYMDEKO (COPACKAGED) | — | 2018-02-12 | fda.gov ↗ |
Trials 43
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 14 trials | ||||||
| Phase 1 | NCT07437105 | Feb 2026 → Jun 2027 expected | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Recruiting | No outcome recorded |
| 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 · 2 missed | ||||||
| Phase 2 | NCT03506061 | Sep 2019 → Feb 2024 | cystic fibrosis | Emory University | Completed | Missed primary |
| Phase 2 | NCT02730208 | Sep 2016 → Jul 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| 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 | Mixed |
| Phase 325 trials · 7 met primary · 1 missed | ||||||
| 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 | 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 | NCT05033080 | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| 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 | NCT04058366 | Dec 2019 → Dec 2022 | 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 | 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 | Met primary |
| 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 | Met primary |
| Phase 3 | NCT03150719 | May 2017 → Aug 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02516410 | Aug 2015 → Jun 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase 3 | NCT02565914 | Aug 2015 → May 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02392234 | Mar 2015 → Feb 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT02347657 | Jan 2015 → Jan 2017 | 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 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 Stop: Business |
| 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 |
| Comparator or background therapy7 trials | ||||||
| Phase 1 | NCT03029455 Background | Nov 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| 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 | Mixed |
| 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 | Met primary |
| 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 | Met primary |
News releases announcing trial results or a regulatory action · 66
| Date | Issuer | Release |
|---|---|---|
| 2026-10-09 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present New Data on ALYFTREK® at the North American Cystic Fibrosis Conference vrtx.com ↗
For decades, pancreatic organ failure and resulting exocrine pancreatic insufficiency has been accepted as an irreversible consequence of CF, but these remarkable data in younger children treated with ALYFTREK show that, in fact, pancreatic failure can be reversed. |
| 2026-06-05 | Vertex Pharmaceuticals Incorporated | Results Vertex Presents New Data on ALYFTREK® at European Cystic Fibrosis Conference vrtx.com ↗
ALYFTREK Phase 3 data on children with cystic fibrosis ages 2 to 5 with vanzacaftor/tezacaftor/deutivacaftor-responsive genotypes including F/F and F/MF shows 65% reached sweat chloride levels of <30 mmol/L |
| 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 ↗
This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. |
| 2025-10-23 | Vertex Pharmaceuticals Incorporated | Results Vertex Presents New Data Across Portfolio of Cystic Fibrosis Medicines Including ALYFTREK® at the North American Cystic Fibrosis Conference vrtx.com ↗
Post-hoc analyses of two pivotal Phase 3 studies (SKYLINE 102 and SKYLINE 103) will be presented, showing that F580del/minimal function mutation (F/MF) subjects treated with ALYFTREK had lower rates of pulmonary exacerbations (PEx), PEx requiring hospitalization or IV antibiotics, |
| 2025-07-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2025-06-06 | Vertex Pharmaceuticals Incorporated | Results Vertex Presents New Data on Benefits of ALYFTREK® and Importance of Achieving Lower Sweat Chloride Levels at the European Cystic Fibrosis Conference vrtx.com ↗
For all clinical outcomes in the study, SwCl levels below 60 mmol/L were associated with greater benefit including better and more stable lung function, fewer pulmonary exacerbations, better nutritional status and better quality of life. |
| 2025-04-28 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis vrtx.com ↗
today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor) |
| 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). |
| 2025-03-07 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today that the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatment |
| 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. |
| 2024-12-20 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces US FDA Approval of ALYFTREK™, a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis (CF) in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK. |
| 2024-09-26 | Vertex Pharmaceuticals Incorporated | Results Vertex Highlights First Oral Presentation of Phase 3 Clinical Data of the Vanza Triple and New Data on Long-Term Impact of TRIKAFTA® at the North American Cystic Fibrosis Conference vrtx.com ↗
Phase 3 data on investigational vanza triple demonstrates non-inferiority to TRIKAFTA® in ppFEV1 and further improvement of CFTR function as measured by sweat chloride |
| 2024-08-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Reports Second Quarter 2024 Financial Results vrtx.com ↗
FDA accepted NDA for vanzacaftor triple in CF with Priority Review and PDUFA target action date of January 2, 2025; additionally, MAA submissions validated in EU and U.K. |
| 2024-07-02 | Vertex Pharmaceuticals Incorporated | Regulatory 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 | Results Vertex Reports Fourth Quarter and Full Year 2023 Financial Results vrtx.com ↗
Vertex today shared positive data from the three pivotal studies for the vanza triple, showing that the studies met the primary endpoint and all key secondary endpoints. |
| 2024-02-05 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis vrtx.com ↗
Treatment with the once-daily vanza triple CFTR modulator regimen met all primary and key secondary endpoints in two randomized controlled trials in people with CF ages 12 years and older |
| 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 ↗ |
| 2021-07-28 | Vertex Pharmaceuticals Incorporated | Results Vertex to Initiate Phase 3 Development Program for New Once-Daily Triple Combination Regimen in People With Cystic Fibrosis vrtx.com ↗
The regimen was generally well-tolerated, and the study met the primary efficacy endpoint of absolute change from baseline in ppFEV1 and all secondary efficacy endpoints including absolute change from baseline in sweat chloride concentration in both patient populations. |
| 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 ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to TRIKAFTA based on in vitro data. |
| 2020-11-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for SYMKEVI® (tezacaftor/ivacaftor) With KALYDECO® (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years vrtx.com ↗
Today’s approval brings us closer to our ultimate goal of providing medicines for all people with CF. |
| 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. |
| 2020-08-21 | 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 Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor) vrtx.com ↗
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the Spanish Government has approved terms for the national reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in combination with KALYDECO® (ivacaftor) for eligible patients in Spain living with cystic fibrosis (CF). |
| 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. |
| 2019-06-04 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present Data at ECFS Conference on Potential Impact of Early Treatment and Long-Term Treatment with CFTR Modulators on the Underlying Cause of CF vrtx.com ↗
lumacaftor/ivacaftor was generally well tolerated for up to 120 weeks, showing a safety profile consistent with previous studies. |
| 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. |
| 2019-03-06 | 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 ↗ |
| 2018-10-18 | 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 ↗
Data from these studies were also published online today in The New England Journal of Medicine (NEJM). |
| 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 |
| 2018-04-26 | Vertex Pharmaceuticals Incorporated | Results Vertex Initiates Phase 3 Studies of VX-445, Tezacaftor and Ivacaftor as a Triple Combination Regimen for People with Cystic Fibrosis vrtx.com ↗
Vertex also today announced safety and efficacy results for the once-daily potentiator, VX-561, when dosed as part of a triple combination regimen with a next-generation corrector (VX-659 or VX-445) and tezacaftor in Phase 2 studies |
| 2017-11-03 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Presentations of Data at North American Cystic Fibrosis Conference that Demonstrate Important Progress Toward Goal of Helping All People with CF vrtx.com ↗
All studies showed statistically significant improvements in lung function (ppFEV1) with a triple combination regimen in these patients. |
| 2017-10-25 | Vertex Pharmaceuticals Incorporated | Results Vertex Reports Third-Quarter 2017 Financial Results vrtx.com ↗
Provides update on clinical development programs, including top-line results for three clinical studies in CF: Phase 3 ORKAMBI in ages 2 to 5; Phase 3 tezacaftor/ivacaftor combination in gating mutations; Phase 2 VX-371 + ORKAMBI in F508del homozygous patients |
| 2017-08-24 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Acceptance of its Applications for Review of the Tezacaftor/Ivacaftor Combination Treatment in People with Cystic Fibrosis by the FDA and EMA vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the acceptance of its applications for the use of the tezacaftor/ivacaftor combination treatment in people with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation or one F508del mutation and one residual function mutation that is responsive to tezacaftor/ivacaftor by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). |
| 2017-07-18 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min) vrtx.com ↗
Initial Phase 2 data also showed mean absolute improvements in ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation (F508del/F508del), who were already receiving tezacaftor and ivacaftor |
| 2017-03-28 | Vertex Pharmaceuticals Incorporated | Results Two Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment Met Primary Endpoints with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis vrtx.com ↗
This study met its primary endpoint with a mean absolute improvement in ppFEV1 through 24 weeks of 4.0 percentage points from baseline compared to placebo (p < 0.0001). |
| 2016-08-15 | Vertex Pharmaceuticals Incorporated | Results Vertex Provides Update on Ongoing Phase 3 Program for VX-661 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis vrtx.com ↗
Vertex plans to stop the study of VX-661 and ivacaftor in people with one copy of the F508del mutation and one copy of a mutation that results in minimal CFTR protein function (F508del het/min). |
| 2015-03-23 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Data from 12-Week Phase 2 Safety Study of VX-661 in Combination with Ivacaftor in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
The study showed that the combination regimen was generally well tolerated, and all patients completed 12 weeks of treatment. |
| 2014-05-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Reports First Quarter 2014 Financial Results and Provides Updates on Key Business Priorities vrtx.com ↗
Vertex also announced today that the U.S. FDA has granted VX-661 Orphan Drug Designation. |
| 2014-05-01 | Vertex Pharmaceuticals Incorporated | Results Addition of VX-661 to KALYDECO® (ivacaftor) Improves Lung Function in People with CF Who Are Heterozygous for the F508del and G551D Mutations in 28-day Phase 2 Proof-of-Concept Study vrtx.com ↗
In addition to improvements in FEV1, the addition of VX-661 also resulted in decreases in sweat chloride through the 28-day treatment period. |
| 2013-04-18 | Vertex Pharmaceuticals Incorporated | Results Treatment with VX-661 and Ivacaftor in a Phase 2 Study Resulted in Statistically Significant Improvements in Lung Function in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
Patients in the 100 and 150 mg combination dose groups showed statistically significant mean relative improvements in lung function, versus placebo, of 9.0 percent (p=0.01) and 7.5 percent (p=0.02), respectively, at Day 28. |
All press releases naming this drug 89 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 54 sources stand behind the page.
| Field | Value | Cited 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 ↗44“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 ↗ |
| 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 ↗ |
| 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 |
| 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 |