Drugs / Vanzacaftor
Vanzacaftor
also known as
VX-121 · VX-121/TEZ/D-IVA · VANZACAFTOR CALCIUM · Vanzacaftor component of alyftrek · Vanzacaftor hemicalcium · VNZ · VX-121 Calcium Salt
Small molecule targets CFTR via restoration
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed cystic fibrosis 6 milestones | |||||
| Filed | EU (EMA) | — | cystic fibrosis | 2024-07-02 | “Vertex also received validation of its vanzacaftor triple Marketing Authorization Application (MAA) submissions from the European Medicines Agency (EMA)” vrtx.com ↗ “Vertex also received validation of its Marketing Authorization Application (MAA) submission by the European Medicines Agency (EMA) in the EU” vrtx.com ↗ |
| Filed | Other | — | cystic fibrosis | 2024-07-02 | “Vertex has also completed regulatory submissions for the vanzacaftor triple in Canada, Australia, New Zealand and Switzerland.” vrtx.com ↗ “The company has also submitted in Canada, Australia, Switzerland and the U.K.” vrtx.com ↗ |
| Filed | UK (MHRA) | — | cystic fibrosis | 2024-07-02 | “The company has also submitted in Canada, Australia, Switzerland and the U.K.” vrtx.com ↗ “additionally, MAA submissions validated in EU and U.K.” vrtx.com ↗ |
| 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 ↗ “FDA accepted NDA for vanzacaftor triple in CF with Priority Review and PDUFA target action date of January 2, 2025” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2024-05-06 | “Submitted NDA and MAA filings for vanzacaftor triple in CF to FDA and EMA, respectively” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2024-05-06 | “Submitted NDA and MAA filings for vanzacaftor triple in CF to FDA and EMA, respectively” vrtx.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Alyftrek | — | 2025-06-30 | europa.eu ↗ |
| Approved | US (FDA) | ALYFTREK | — | 2024-12-20 | fda.gov ↗ |
Trials 10
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 14 trials | ||||||
| Phase 1 | NCT06299709 | Mar → May 2024 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT05867147 | Apr → Jun 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT05535959 | Sep → Nov 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT05437120 | Jul 2022 → Mar 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03768089 | Mar 2018 → May 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT03912233 | Apr → Dec 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 34 trials · 2 met primary | ||||||
| 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 | NCT05076149 | Oct 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT05033080 | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
News releases announcing trial results or a regulatory action · 15
| 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) |
| 2025-03-07 | 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 |
| 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 |
| 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. |
All press releases naming this drug 16 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Vanzacaftor | “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 20253“vanzacaftor (20 mg once daily), tezacaftor (100 mg once daily), and deutivacaftor (250 mg once daily) as two fixed-dose combination tablets in the morning” PMID 39756424 ↗ Jan 2025 “Vanzacaftor/tezacaftor/deutivacaftor” NCT06299709 ↗ |
| Known as | VANZACAFTOR CALCIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314934 ↗ |
| Known as | Vanzacaftor component of alyftrek | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314934 ↗ |
| Known as | Vanzacaftor hemicalcium | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314934 ↗ |
| Known as | VNZ | “The purpose of this study is to evaluate the effect of Vanzacaftor (VNZ) on QTcF, as well as the pharmacokinetic (PK), safety, and tolerability of VNZ in healthy participants.” NCT05867147 ↗ |
| Known as | VX-121 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03912233 ↗ |
| Known as | VX-121 Calcium Salt | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314934 ↗ |
| Known as | VX-121/TEZ/D-IVA | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05076149 ↗ |
| Action | Restore | “Vanzacaftor-tezacaftor-deutivacaftor restored CFTR function in vitro” PMID 39756424 ↗ Jan 2025 |
| Modality | Small molecule | — CHEMBL5314934 ↗ |
| Route | Oral | “Suspension for oral administration.” NCT03768089 ↗ |
| Target | CFTR | “Vanzacaftor-tezacaftor-deutivacaftor restored CFTR function in vitro” PMID 39756424 ↗ Jan 2025 |