Drugs / deutivacaftor
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 12
| 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 | NCT06154447 | Dec 2023 → Jul 2026 overdue | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Active not recruiting | No outcome recorded |
| Phase 1 | NCT02680249 | Feb → Apr 2016 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02599792 | Nov 2015 → Apr 2016 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03227471 | Jan 2017 → Mar 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 24 trials · 1 met primary | ||||||
| Phase 2 | NCT03912233 | Apr → Dec 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT03911713 | Apr 2019 → Aug 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT03224351 | Aug 2017 → Feb 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 2 | NCT02971839 | Dec 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase 33 trials · 1 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 | NCT05033080 | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT07437105 Background | Feb 2026 → Jun 2027 expected | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| 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. |
| 2026-04-01 | 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. |
| 2025-07-01 | 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. |
| 2025-04-28 | 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 |
| 2024-08-01 | 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 13 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Deutivacaftor | “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 20255“The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.” NCT05444257 ↗ “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 | CTP-656 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02680249 ↗ |
| Known as | VX-561 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03224351 ↗ |
| Route | Oral | “CTP-656 solid oral doses” NCT02599792 ↗ |