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 · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Alyftrek | — | 2025-06-30 | europa.eu ↗ |
| Approved | US (FDA) | ALYFTREK | — | 2024-12-20 | fda.gov ↗ |
Trials 10 · a red edge is where a trial was stopped
| 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 | ||||||
| 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 | No outcome recorded |
| Phase 3 | NCT05033080 | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
Press releases naming this drug 4 releases
| Date | Issuer | Release |
|---|---|---|
| 2025-07-14 | Vertex Pharmaceuticals Incorporated | Vertex Announces Broad Reimbursement Agreement With NHS England for ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor) an Innovative Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis vrtx.com ↗ |
| 2025-03-07 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2024-07-02 | Vertex Pharmaceuticals Incorporated | 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 | Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis vrtx.com ↗ |
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 NCT05437120 ↗ |
| 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 |