Drugs / Tezacaftor/Ivacaftor
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | SYMDEKO | cystic fibrosis | 2020-09-01 | “today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and...” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2019-04-30 | “In late 2018, sNDAs were submitted to the FDA for ivacaftor in infants ages 6 to <12 months and for tezacaftor/ivacaftor in children ages 6 through 11.” vrtx.com ↗ “- Supplemental New Drug Application (sNDA) submitted in the U.S. for tezacaftor/ivacaftor in children ages 6 to 11 years old.” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2017-07-26 | “has submitted a new drug application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the tezacaftor/ivacaftor” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2017-07-26 | “has submitted a new drug application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the tezacaftor/ivacaftor” vrtx.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 3 | NCT02412111 | Jun 2015 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 41
| 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. |
| 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. |
| 2024-09-26 | 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 ↗
Real-world evidence and clinical studies of TRIKAFTA® continue to show sustained long-term benefits including improvement in pancreatic function in young patients |
| 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.” |
| 2022-04-20 | 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. |
| 2021-10-19 | 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. |
| 2021-03-24 | 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. |
| 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. |
| 2020-11-27 | 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. |
| 2020-09-10 | 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). |
| 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 ↗ |
| 2019-10-19 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and ORKAMBI® (lumacaftor/ivacaftor) in Children Ages 2 to 5, With Certain CFTR Mutations in Australia vrtx.com ↗
People with CF who have one copy of the F508del mutation and another responsive RF mutation in the CFTR gene will have access to a medicine for the cause of their CF for the first time. |
| 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. |
| 2019-03-12 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Approval for SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) in Australia, to Treat the Underlying Cause of Cystic Fibrosis in People aged 12 and Older with Certain CFTR Gene Mutations 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 ↗ |
| 2019-02-14 | Vertex Pharmaceuticals Incorporated | Results Positive Phase 3 Study for Tezacaftor/Ivacaftor Combination in Children Aged 6-11 Years with Cystic Fibrosis Supports European Medicines Agency Submission vrtx.com ↗
The study met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 8 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among patients treated with tezacaftor/ivacaftor. |
| 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 ↗ |
| 2018-11-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene vrtx.com ↗
the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) |
| 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 ↗
Patient-reported outcomes data from Phase 3 EVOLVE and EXPAND studies continue to support the clinical benefit of SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) |
| 2018-07-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for SYMKEVI® (tezacaftor/ivacaftor) for People with Cystic Fibrosis Aged 12 and Older with Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals (Europe) Limited, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. |
| 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 |
| 2018-06-07 | Vertex Pharmaceuticals Incorporated | Results Vertex Data Presented at European Cystic Fibrosis Society (ECFS) Conference Demonstrate Early and Long-Term Disease-Modifying Potential of Treating the Underlying Cause of CF vrtx.com ↗
Interim analysis of the ongoing extension study of tezacaftor/ivacaftor combination (approved in the U.S. as SYMDEKOTM) continues to demonstrate consistent safety and sustained benefits up to 48 weeks |
| 2018-02-12 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves SYMDEKOTM (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 the U.S. Food and Drug Administration (FDA) approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. |
| 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 ↗
Safety data from this interim analysis showed that tezacaftor/ivacaftor was generally well tolerated and had a safety profile consistent with that seen in EVOLVE and EXPAND. |
| 2017-11-03 | Vertex Pharmaceuticals Incorporated | Results Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment in People with Cystic Fibrosis Ages 12 and Older Published in the New England Journal of Medicine vrtx.com ↗
In both studies, the tezacaftor/ivacaftor combination demonstrated statistically significant and clinically meaningful improvements in lung function and other measures of disease. |
| 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). |
All press releases naming this drug 49 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Tezacaftor/Ivacaftor | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02412111 ↗ |
| Known as | TEZ/IVA | “Tezacaftor (TEZ)/ivacaftor (IVA) is an approved CFTR modulator shown to be efficacious and generally safe and well tolerated in people ≥12 years of age with cystic fibrosis...” PMID 33339768 ↗ Dec 2020 |
| Route | Oral | “VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning” NCT02412111 ↗ |
| Target | CFTR | “Tezacaftor (TEZ)/ivacaftor (IVA) is an approved CFTR modulator” PMID 33339768 ↗ Dec 2020 |