Drugs / Ivacaftor
Regulatory milestones approvals, filings & regulatory actions · 28 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed cystic fibrosis 15 milestones | |||||
| Filed | EU (EMA) | KAFTRIO | cystic fibrosis | 2020-09-14 | “EMA has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of KAFTRIO®* (ivacaftor/tezacaftor/elexacaftor)” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2019-10-31 | “Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen.” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2019-07-22 | “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.” vrtx.com ↗ |
| Filed | US (FDA) | — | cystic fibrosis | 2018-04-26 | “Submission of supplemental New Drug Application (sNDA) of ivacaftor in children ages 12 to <24 months” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2018-04-26 | “a Marketing Authorization Application (MAA) line extension for ivacaftor in this age group has been submitted to the European Medicines Agency (EMA)” vrtx.com ↗ |
| Filed | EU (EMA) | — | cystic fibrosis | 2018-01-31 | “The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the tezacaftor/ivacaftor combination.” vrtx.com ↗ “The EMA has validated the MAA for the tezacaftor/ivacaftor combination” vrtx.com ↗ |
| Rejected (CRL) | US (FDA) | KALYDECO | cystic fibrosis | 2016-02-05 | “Vertex submitted an sNDA for approval of KALYDECO for treatment of people with CF ages 2 and older who have one of 23 residual function mutations and received a Complete Response Letter” vrtx.com ↗ “Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO® (ivacaftor)” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2015-10-07 | “FDA has accepted for review a supplemental New Drug Application (sNDA) for the use of KALYDECO® (ivacaftor)” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2014-10-09 | “Vertex today announced the submission of an NDA in the United States for the approval of ivacaftor in children with CF ages 2 to 5” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2013-12-19 | “In December 2013, the U.S. Food and Drug Administration (FDA) accepted Vertex's supplemental New Drug Application (sNDA) for ivacaftor” vrtx.com ↗ “Vertex today announced that the U.S. FDA accepted its sNDA for ivacaftor and granted Vertex's request for six-month Priority Review.” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2013-09-30 | “today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA)” vrtx.com ↗ |
| Filed | Other | KALYDECO | cystic fibrosis | 2012-11-01 | “Vertex has submitted marketing applications for KALYDECO in Canada and Australia” vrtx.com ↗ “Vertex has submitted marketing applications for KALYDECO in Canada” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2011-12-15 | “the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECO” vrtx.com ↗ |
| Filed | EU (EMA) | KALYDECO | cystic fibrosis | 2011-12-15 | “Last week, Vertex submitted an NDA to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for KALYDECO” vrtx.com ↗ “Vertex submitted an sNDA in the U.S. and MAA variation in Europe for approval of ivacaftor in people with the R117H mutation.” vrtx.com ↗ “its marketing authorization application (MAA) for KALYDECO has been validated by the European Medicines Agency (EMA)” vrtx.com ↗ |
| Filed | US (FDA) | KALYDECO | cystic fibrosis | 2011-10-19 | “Last week, Vertex submitted an NDA to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for KALYDECO” vrtx.com ↗ “today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for KALYDECO™ (VX-770, ivacaftor)” vrtx.com ↗ “Vertex submitted an sNDA in the U.S. and MAA variation in Europe for approval of ivacaftor in people with the R117H mutation.” 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 ↗ “In late 2018, Vertex submitted an sNDA to the U.S. Food and Drug Administration (FDA) for tezacaftor/ivacaftor” vrtx.com ↗ “KALYDECO in people ages 2 and older with one of 23 residual function mutations was accepted for review” vrtx.com ↗ “In late 2018, sNDAs were submitted to the FDA for ivacaftor in infants ages 6 to <12 months” vrtx.com ↗ “In October 2015, Vertex submitted an sNDA for approval of KALYDECO” vrtx.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (11) | US (FDA) | KALYDECO | — | 2014-02-21 – 2025-05-22 | fda.gov ↗ |
| Approved | EU (EMA) | Kalydeco | — | 2012-07-23 | europa.eu ↗ |
| Approved | US (FDA) | KALYDECO | — | 2012-01-31 | fda.gov ↗ |
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| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 02 trials | ||||||
| Phase 0 | NCT03439865 | May 2019 → Apr 2027 expected | chronic rhinosinusitis | University of Alabama at Birmingham | Recruiting | No outcome recorded |
| Phase 0 | NCT02135432 | May → Oct 2015 | chronic obstructive pulmonary disease | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 111 trials | ||||||
| Phase 1 | NCT02015507 | Jan → Feb 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01899105 | Jul → Aug 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01910415 | Jun 2013 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01888393 | Jun → Nov 2013 | — | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01768663 | Jan → Sep 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01216046 | Oct 2010 → May 2011 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01208285 | Sep → Dec 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01153542 | Jun → Aug 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01060566 | Feb → Mar 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT01018368 | Nov 2009 → Mar 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 1 | NCT00966602 | Sep → Dec 2009 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 217 trials · 3 met primary · 5 missed | ||||||
| Phase 2 | NCT04581408 | Jun 2021 → Apr 2025 | long QT syndrome | Istituto Auxologico Italiano | Completed | No outcome recorded |
| Phase 2 | NCT04135495 | Nov 2019 → Oct 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04126473 | Nov 2019 → Apr 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT03506061 | Sep 2019 → Feb 2024 | cystic fibrosis | Emory University | Completed | Missed primary |
| Phase 2 | NCT03625466 | Aug 2018 → Oct 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase 2 | NCT03061331 | Jan → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase 2 | NCT02730208 | Sep 2016 → Jul 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase 2 | NCT02871778 | Aug 2016 → Nov 2018 | primary ciliary dyskinesia | Parion Sciences | Completed | Met 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 | NCT01685801 | Sep 2012 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 2 | NCT01531673 | Feb 2012 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Mixed |
| Phase 2 | NCT01262352 | Jan → Nov 2011 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 2 | NCT01225211 | Oct 2010 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01161537 | Oct 2010 → Feb 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT00953706 | Sep 2009 → Jul 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | Missed primary |
| Phase 2 | NCT00457821 | May 2007 → Aug 2008 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 346 trials · 14 met primary · 3 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 | 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 | 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 | NCT04235140 | Feb 2020 → Aug 2023 | 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 | NCT04105972 | Oct 2019 → Jul 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| 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 | NCT03277196 | Aug 2017 → Oct 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT03068312 | Jul 2017 → Dec 2018 | 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 | NCT03125395 | May 2017 → Jul 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02934698 | Nov 2016 → May 2018 | cystic fibrosis | Medical University of South Carolina | Completed | No outcome recorded |
| Phase 3 | NCT02725567 | Jun 2016 → Jun 2022 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT02742519 | May 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | Missed primary Stop: Enrollment |
| Phase 3 | NCT02797132 | May 2016 → Sep 2017 | 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 | NCT02544451 | Aug 2015 → Aug 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02565914 | Aug 2015 → May 2019 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02514473 | Jul 2015 → Sep 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT02412111 | Jun 2015 → Sep 2017 | 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 | NCT02390219 | Mar 2015 → Oct 2016 | cystic fibrosis, lung disorder | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02347657 | Jan 2015 → Jan 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT01946412 | Dec 2013 → Dec 2015 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01931839 | Oct 2013 → Apr 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01897233 | Jul 2013 → Oct 2015 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT01807923 | May 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT01807949 | Apr 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT01707290 | Feb 2013 → Apr 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01705145 | Jan 2013 → Mar 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01614457 | Jul 2012 → Oct 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase 3 | NCT01614470 | Jul 2012 → Oct 2013 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT01117012 | Jul 2010 → May 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT00909727 | Aug 2009 → Nov 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT00909532 | Jun 2009 → Jul 2010 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 410 trials · 1 met primary · 1 missed | ||||||
| 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 4 | NCT03251911 | Jan 2020 → Feb 2022 | chronic bronchitis | University of Alabama at Birmingham | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 4 | NCT03624101 | Dec 2018 → Oct 2019 | cystic fibrosis | University of Alabama at Birmingham | Terminated | No outcome recorded |
| Phase 4 | NCT03956589 | Oct 2018 → Apr 2020 | cystic fibrosis | University Hospital, Antwerp | Terminated | No outcome recorded |
| Phase 4 | NCT03256799 | Mar → Jul 2017 | cystic fibrosis | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 4 | NCT02875366 | Sep 2016 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| Phase not applicable6 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 |
| — | NCT02653027 | Jan → Feb 2018 | cystic fibrosis, diabetes mellitus | Massachusetts General Hospital | Withdrawn | No outcome recorded |
| — | NCT02858843 | Aug 2016 → May 2018 | cystic fibrosis, diabetes mellitus | Massachusetts General Hospital | Terminated | No outcome recorded Stop: Enrollment |
| — | NCT02310789 | Jul 2015 → Aug 2016 | cystic fibrosis | Richard Barry Moss | Completed | No outcome recorded |
| — | NCT01784419 | Oct 2013 → Dec 2015 | cystic fibrosis | University of California, San Francisco | Completed | No outcome recorded |
| Phase not stated3 trials | ||||||
| — | NCT01381289 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
| — | NCT03278314 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
| — | NCT04058210 | — | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Approved for marketing | No outcome recorded |
| Comparator or background therapy17 trials | ||||||
| Phase 1 | NCT03029455 Background | Nov 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 1 | NCT02599792 Comparator | Nov 2015 → Apr 2016 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02392702 Comparator | Mar → Jun 2015 | — | Concert Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT06237335 Background | Feb 2024 → Aug 2026 overdue | cystic fibrosis | ReCode Therapeutics | Recruiting | No outcome recorded |
| Phase 2 | NCT04066751 Comparator | Dec 2021 → Oct 2024 | chronic obstructive pulmonary disease | University of Alabama at Birmingham | Withdrawn | No outcome recorded |
| Phase 2 | NCT04006873 Comparator | Sep 2019 → Oct 2020 | cystic fibrosis | Nottingham University Hospitals NHS Trust | Completed | No outcome recorded |
| Phase 2 | NCT03085485 Comparator | Mar 2017 → Sep 2022 | chronic bronchitis | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 2 | NCT02951195 Background | Nov 2016 → Jan 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Mixed |
| Phase 3 | NCT05033080 Comparator | Sep 2021 → May 2023 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT04183790 Background | Feb 2020 → Feb 2024 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT04058353 Background | Aug 2019 → Jun 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 3 | NCT03525548 Background | 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 Background | May → Sep 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Met primary |
| Phase 4 | NCT02823470 Background | Jun 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded Stop: Enrollment |
| Phase 4 | NCT01937325 Comparator | Feb → Dec 2014 | cystic fibrosis | The Alfred | Unknown | No outcome recorded |
| — | NCT03894657 Background | Dec 2019 → Nov 2021 | cystic fibrosis | Assistance Publique - Hôpitaux de Paris | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 132
| 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. |
| 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). |
| 2025-02-28 | 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 ↗ |
| 2025-01-09 | Lupin Limited | Regulatory Lupin Receives Tentative Approval from U.S. FDA for Ivacaftor Oral Granules lupin.com ↗
it has received tentative approval from the United States Food and Drug Administration (U.S. FDA) for its Abbreviated New Drug Application for Ivacaftor Oral Granules |
| 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 ↗
Vertex will have four poster presentations that include clinical trial and real-world evidence data, three specifically on TRIKAFTA® and one showing sustained benefits from KALYDECO®, which is approved for treatment in the youngest ages. |
| 2024-04-26 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KALYDECO® to Treat Infants With Cystic Fibrosis Ages 1 Month and Older vrtx.com ↗
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KALYDECO® (ivacaftor) for the treatment of infants down to 1 month of age with cystic fibrosis (CF) who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. |
| 2024-02-23 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for KALYDECO® for the Treatment of Infants With Cystic Fibrosis Ages 1 Month and Older vrtx.com ↗ |
| 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 ↗ |
| 2023-07-05 | Vertex Pharmaceuticals Incorporated | Regulatory European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old vrtx.com ↗
Vertex Pharmaceuticals today announced that the European Commission has granted approval for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to <2 years old |
| 2023-05-03 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than four months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO® based on clinical and/or in vitro assay data. |
| 2023-04-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old 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. |
| 2022-09-02 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of ORKAMBI® (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) ages 12 to <24 months who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 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 ↗
Long-term real-world study results show significant benefits of initiating KALYDECO® (ivacaftor) at young age |
| 2022-06-08 | Lupin Limited | Regulatory Lupin Receives Tentative Approval from U.S. FDA for Ivacaftor Tablets lupin.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 ↗
Real-world data from people treated with KALYDECO® (ivacaftor) over approximately 6 years show lower rates of mortality, lung transplant and pulmonary exacerbations than comparator cohort |
| 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. |
| 2021-03-26 | 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. |
| 2020-11-27 | 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. |
| 2020-11-05 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age vrtx.com ↗ |
| 2020-09-25 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO® (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With CF as Early as Four Months of Age vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) for use in children with cystic fibrosis (CF) ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO based on clinical and/or in vitro assay data. |
| 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 ↗ |
| 2020-06-10 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying CF. |
| 2020-05-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages 6 Months and 18 Years With the R117H Mutation in the CFTR Gene vrtx.com ↗
the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KALYDECO® (ivacaftor) |
| 2019-12-10 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) in Infants With Cystic Fibrosis Ages 6 Months to Less Than 12 Months With Certain Mutations in the CFTR Gene vrtx.com ↗
For the first time, children with CF in Europe as young as 6 months with certain mutations now have access to a medicine that treats the underlying cause of their disease. |
| 2019-11-20 | Vertex Pharmaceuticals Incorporated | Regulatory French Authorities Approve National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) for Eligible People Ages Two and Older With Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the French Authorities (Comité économique des produits de santé, or CEPS) have approved national reimbursement of ORKAMBI® (lumacaftor/ivacaftor) for people ages two and older with cystic fibrosis (CF) who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 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. |
| 2019-10-31 | Vertex Pharmaceuticals Incorporated | Results Phase 3 Results from Two Studies of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New England Journal of Medicine and The Lancet vrtx.com ↗
Both studies met primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful improvements in lung function and other measures of disease. |
| 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 ↗ |
| 2019-10-18 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) to Treat Eligible Infants with Cystic Fibrosis as Early as 6 Months of Age 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. |
| 2019-01-28 | Vertex Pharmaceuticals Incorporated | Regulatory Health Canada Grants Market Authorization for KALYDECO® (ivacaftor) in Children Ages 12 to <24 months with Certain Mutations in the CFTR Gene vrtx.com ↗ |
| 2019-01-21 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease vrtx.com ↗ |
| 2018-12-13 | Vertex Pharmaceuticals Incorporated | Regulatory Health Canada Grants Market Authorization for ORKAMBI® (lumacaftor/ivacaftor) for Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced that Health Canada has granted Market Authorization for ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del CFTR mutation. |
| 2018-11-29 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) to Treat Patients with Cystic Fibrosis Aged 12 to <24 months with Certain Mutations in the CFTR Gene vrtx.com ↗ |
| 2018-11-16 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease 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 ORKAMBI® (lumacaftor/ivacaftor) |
| 2018-10-19 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for KALYDECO® (ivacaftor) to Treat Patients With Cystic Fibrosis Aged 12 to <24 months With Certain Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals (Europe) Limited today announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KALYDECO® (ivacaftor) to include the treatment of people with cystic fibrosis (CF) aged 12 to <24 months who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. |
| 2018-09-03 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Reimbursement Agreement in Australia for ORKAMBI® (lumacaftor/ivacaftor) for People with Cystic Fibrosis Ages Six Years and Older with Two Copies of the F508del Mutation vrtx.com ↗
This agreement means lumacaftor/ivacaftor will be listed on the Pharmaceutical Benefits Scheme (PBS) from 1 October and follows a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC). |
| 2018-08-20 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives a Positive PBAC Recommendation for Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) to Treat Australians Ages Six and Over with Cystic Fibrosis and Two Copies of the F508del Mutation vrtx.com ↗
We welcome the PBAC decision and are now working with the Australian Government to finalize the agreement as quickly as possible to make lumacaftor/ivacaftor available to patients. |
| 2018-01-10 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation, to include children ages 6 through 11. |
| 2017-12-07 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Results from Open-Label Phase 3 Study of KALYDECO® (ivacaftor) in Children with Cystic Fibrosis Ages 1 to 2 Years vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from an open-label Phase 3 study of KALYDECO® (ivacaftor) in children with cystic fibrosis (CF) ages 1 to 2 years who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2017-11-10 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation in the European Union vrtx.com ↗ |
| 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 ↗
Data from ongoing extension study of ORKAMBI® (lumacaftor/ivacaftor) in children ages 6-11 and real-world KALYDECO® (ivacaftor) data demonstrate long-term safety and other benefits of these medicines |
| 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 ↗
the ivacaftor monotherapy group demonstrating a mean absolute improvement of 4.7 percentage points compared to placebo (p < 0.0001). |
| 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). |
| 2017-08-01 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO® (ivacaftor) for More Than 600 People Ages 2 and Older With Cystic Fibrosis Who Have Certain Residual Function Mutations vrtx.com ↗
the U.S. Food and Drug Administration (FDA) has approved KALYDECO® (ivacaftor) for use in more than 600 people with cystic fibrosis (CF) ages 2 and older |
| 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 |
| 2017-07-13 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Reimbursement Agreement in Italy for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation vrtx.com ↗
Regional authorities will now begin implementation to provide the hundreds of eligible patients in Italy access to this important medicine. |
| 2017-06-09 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Nine Presentations of Data on ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) at the European Cystic Fibrosis Society (ECFS) Conference vrtx.com ↗
The data presented at the Conference demonstrate that treating the underlying cause of CF with CFTR modulators can modify the progression of the disease. |
| 2017-05-17 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO® (ivacaftor) for More Than 900 People Ages 2 and Older with Cystic Fibrosis Who Have Certain Residual Function Mutations vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved KALYDECO® (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 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). |
| 2016-11-07 | Vertex Pharmaceuticals Incorporated | Results Positive Phase 3 Study of ORKAMBI® in Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies of the F508del Mutation Supports a Submission to the European Medicines Agency in the First Half of 2017 vrtx.com ↗
The study met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among patients treated with ORKAMBI compared to placebo. |
| 2016-10-27 | Vertex Pharmaceuticals Incorporated | Results Vertex Presents Long-Term Data Demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) Show the Potential to Modify the Progression of CF vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the presentation of long-term data demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) show the potential to modify the progression of cystic fibrosis (CF). |
| 2016-10-27 | Lakefront Biotherapeutics NV | Results Galapagos to present progress in CF programs at NACFC 2016 lakefrontbio.com ↗
The novel potentiator GLPG1837 modulates CFTR through different mode of action than ivacaftor (Kalydeco® ). |
| 2016-09-28 | Vertex Pharmaceuticals Incorporated | Regulatory U.S. Food and Drug Administration Approves ORKAMBI® (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved ORKAMBI® (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. |
| 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). |
| 2016-06-10 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Presentations of Data for KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) at European Cystic Fibrosis Society (ECFS) Conference vrtx.com ↗
Real-world data presented at ECFS show long-term impact of KALYDECO across multiple measures of disease |
| 2016-05-31 | Vertex Pharmaceuticals Incorporated | Regulatory U.S. FDA Accepts for Priority Review Supplemental New Drug Application for the Use of ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6 to 11 who have Two Copies of the F508del Mutation vrtx.com ↗
The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act (PDUFA). |
| 2016-03-08 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Australian Approval for ORKAMBI® (lumacaftor/ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation vrtx.com ↗
Today's approval is an important step toward making ORKAMBI available for eligible Australians who do not currently have a medicine to treat the underlying cause of their disease. |
| 2016-02-05 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Complete Response Letter from U.S. FDA for Use of KALYDECO® (ivacaftor) in People with Cystic Fibrosis Ages 2 and Older with One of 23 Residual Function Mutations vrtx.com ↗ |
| 2016-01-26 | Vertex Pharmaceuticals Incorporated | Regulatory Health Canada Approves PrORKAMBI® (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has approved PrORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. |
| 2015-11-20 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation vrtx.com ↗ |
| 2015-11-18 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Two EU Approvals for KALYDECO® (ivacaftor) for People with Cystic Fibrosis vrtx.com ↗
European Commission approves expanded use of ivacaftor in children with cystic fibrosis ages 2 to 5 who have one of 9 gating mutations |
| 2015-10-07 | Vertex Pharmaceuticals Incorporated | Regulatory Supplemental New Drug Application for Use of KALYDECO® (ivacaftor) in People with Cystic Fibrosis Ages 2 and Older who have One of 23 Residual Function Mutations Accepted for Priority Review by U.S. FDA vrtx.com ↗
The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of February 6, 2016 was set under the Prescription Drug User Fee Act (PDUFA) for the FDA's decision on the sNDA. |
| 2015-09-25 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives CHMP Positive Opinions for ORKAMBI™ (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) in the European Union vrtx.com ↗
CHMP recommends Marketing Authorization for ivacaftor for children with cystic fibrosis ages 2 to 5 with 9 gating mutations & for people with cystic fibrosis ages 18 and older with the R117H mutation |
| 2015-07-02 | Vertex Pharmaceuticals Incorporated | Regulatory ADDING MULTIMEDIA FDA Approves ORKAMBI™ (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved ORKAMBI™ (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. |
| 2015-06-11 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Data Presentations at European Cystic Fibrosis Society (ECFS) Conference vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from PROGRESS, the long-term extension study of the investigational regimen ORKAMBI™ (lumacaftor/ivacaftor). |
| 2015-05-17 | Vertex Pharmaceuticals Incorporated | Results New England Journal of Medicine Publishes Data from Two Phase 3 Studies of ORKAMBITM (lumacaftor/ivacaftor) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the New England Journal of Medicine (NEJM) published data from the two Phase 3 studies of ORKAMBITM (lumacaftor/ivacaftor), an investigational medicine designed to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation, the most common form of the disease. |
| 2015-05-12 | Vertex Pharmaceuticals Incorporated | Regulatory Food and Drug Administration Advisory Panel Voted 12 to 1 to Recommend Approval of ORKAMBI™ (lumacaftor/ivacaftor) to Treat People with Cystic Fibrosis Ages 12 and Older Who Have Two Copies of the F508del Mutation vrtx.com ↗ |
| 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. |
| 2015-03-18 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives U.S. Food and Drug Administration Approval of KALYDECO® (ivacaftor) for Children with Cystic Fibrosis Ages 2 to 5 who have Specific Mutations in the CFTR Gene vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved KALYDECO® for use in children ages 2 to 5 with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H). |
| 2014-12-29 | Vertex Pharmaceuticals Incorporated | Regulatory U.S. Food and Drug Administration Approves KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved a supplemental new drug application (sNDA) for the use of KALYDECO® (ivacaftor) in people with cystic fibrosis (CF) ages 6 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2014-11-05 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗ |
| 2014-10-21 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation vrtx.com ↗
"Today's recommendation is a positive and important step toward making ivacaftor available for people ages 6 and older with the R117H mutation," said Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex. |
| 2014-10-09 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Reviews Recent Progress and Announces Upcoming Milestones in the Development of Multiple Combinations of Medicines that Target the Underlying Cause of Cystic Fibrosis vrtx.com ↗
New Drug Application submitted for approval of ivacaftor in children ages 2 to 5 with specific mutations in the CFTR gene |
| 2014-07-31 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European Approval for KALYDECO™ (ivacaftor) in Eight Non-G551D Gating Mutations vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has approved KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2014-06-30 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration for Use of KALYDECO® (ivacaftor) in People 18 and Older with Cystic Fibrosis who have the R117H Mutation vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of KALYDECO® in people with cystic fibrosis (CF) ages 18 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2014-06-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for KALYDECO™ (ivacaftor) in Eight Non-G551D Gating Mutations vrtx.com ↗
Today's recommendation for the use of ivacaftor in people with eight additional gating mutations represents an important step toward our goal of helping more people with CF. |
| 2014-06-24 | Vertex Pharmaceuticals Incorporated | Results Two 24-Week Phase 3 Studies of Lumacaftor in Combination with Ivacaftor Met Primary Endpoint with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation vrtx.com ↗
All four 24-week combination treatment arms in the studies, known as TRAFFIC and TRANSPORT, met their primary endpoint of mean absolute improvement in ppFEV1 from baseline compared to placebo at the end of treatment. |
| 2014-06-12 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Presentation of New KALYDECO™ (ivacaftor) Data at European Cystic Fibrosis Society Conference vrtx.com ↗
New data from an analysis of Phase 3 and long-term extension studies in people with cystic fibrosis (CF) who have the G551D mutation showed that treatment with KALYDECO reduced the annual loss of lung function by half over three years compared to similar untreated CF patients. |
| 2014-06-04 | Vertex Pharmaceuticals Incorporated | Results Proof-of-Concept Study of Ivacaftor Monotherapy Showed Improvements in Lung Function After Two Weeks of Treatment in People with Cystic Fibrosis who have a Residual Function Mutation vrtx.com ↗
In part one, a statistically significant improvement in mean absolute lung function (percent predicted forced expiratory volume in one second; PPFEV1) was observed after treatment with ivacaftor for two weeks compared with placebo. |
| 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. |
| 2014-02-21 | Vertex Pharmaceuticals Incorporated | Regulatory U.S. Food and Drug Administration Approves KALYDECO™ (ivacaftor) for Use in Eight Additional Mutations that Cause Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 2013-12-19 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Results of Phase 3 Study of Ivacaftor in People with CF who have the R117H Mutation vrtx.com ↗
The study did not meet its primary endpoint of the absolute change from baseline in FEV1 (percent predicted forced expiratory volume in one second, FEV1) throughout the treatment period for ivacaftor compared to placebo across all patients. |
| 2013-09-30 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration for KALYDECO™ (ivacaftor) Monotherapy for People with Non-G551D Gating Mutations vrtx.com ↗
today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) |
| 2013-07-29 | Vertex Pharmaceuticals Incorporated | Results Results from Phase 3 Study of Ivacaftor Monotherapy Showed Statistically Significant Improvements in Lung Function in People with non-G551D Gating Mutations vrtx.com ↗ |
| 2013-07-09 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Australian Approval for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People with a Specific Genetic Mutation (G551D) vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods Administration (TGA) of Australia has approved KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. |
| 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. |
| 2013-01-06 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Outlines Corporate Strategy and Defines Key 2013 Business Priorities vrtx.com ↗
First two Breakthrough Therapy Designations from U.S. FDA granted to ivacaftor monotherapy and to the combination regimen of VX-809 and ivacaftor for the treatment of cystic fibrosis |
| 2012-12-03 | Vertex Pharmaceuticals Incorporated | Regulatory Health Canada Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People with a Specific Genetic Mutation (G551D) vrtx.com ↗ |
| 2012-09-25 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Presentation of Data at North American Cystic Fibrosis Conference vrtx.com ↗
The Investigational CFTR Corrector, VX-809 (Lumacaftor) Co-Administered with the Oral Potentiator Ivacaftor Improved CFTR and Lung Function in F508DEL Homozygous Patients: Phase II Study Results. |
| 2012-07-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European Approval for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People With a Specific Genetic Mutation (G551D) vrtx.com ↗
The approval of KALYDECO comes two months after the European Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion and is the first European approval for Vertex. |
| 2012-06-28 | Vertex Pharmaceuticals Incorporated | Results Final Data from Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) Showed Statistically Significant Improvements in Lung Function in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
When patients received the two medicines in combination (Day 28 to 56), there was a statistically significant improvement in lung function (percent predicted forced expiratory volume in one second, FEV1) in each of the homozygous treatment groups compared to placebo. |
| 2012-06-07 | Vertex Pharmaceuticals Incorporated | Results Data From Follow-Up Study of KALYDECO™ (ivacaftor) Showed Durable Improvements in Lung Function and Other Measures of Disease in People with Cystic Fibrosis Who Have a Specific Genetic Mutation (G551D) vrtx.com ↗
were durable for up to 96 total weeks of treatment. |
| 2012-05-29 | Vertex Pharmaceuticals Incorporated | Results Vertex Corrects and Provides Additional Data from Recent Interim Analysis of Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
Patients treated with VX-809 and KALYDECO experienced an 8.5 percentage point mean absolute improvement in lung function compared to patients treated with placebo (p=0.002) |
| 2012-05-25 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis vrtx.com ↗ |
| 2012-05-07 | Vertex Pharmaceuticals Incorporated | Results Interim Data from Phase 2 Combination Study of VX-809 and KALYDECO™ (ivacaftor) Showed Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
There was a statistically significant improvement in lung function (absolute change in percent predicted FEV1) across the combined treatment groups relative to baseline compared to placebo (p=0.002). |
| 2012-01-31 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved KALYDECOTM (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), a rare, genetic disease. |
| 2011-12-15 | Vertex Pharmaceuticals Incorporated | Regulatory FDA Grants Priority Review for KALYDECO™ (ivacaftor), the First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECOTM (ivacaftor) and granted the company's request for six-month Priority Review. |
| 2011-11-03 | Vertex Pharmaceuticals Incorporated | Results Phase 3 Study of KALYDECO™ (ivacaftor) in Children Ages 6 to 11 with a Specific Type of Cystic Fibrosis Showed Significant Improvements in Lung Function and Other Measures of Disease Sustained Through 48 Weeks vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced final results from the ENVISION study, the second of two pivotal Phase 3 studies of KALYDECO™ (ivacaftor, VX-770), a medicine in development that targets the defective protein that causes cystic fibrosis (CF). |
| 2011-11-03 | Vertex Pharmaceuticals Incorporated | Results Data from the First Part of a Phase 2 Study Support the Approach of Treating the Most Common Form of Cystic Fibrosis (F508del) by Targeting the Underlying Cause of the Disease With a Combination of KALYDECO™ (ivacaftor) and VX-809 vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from the first part of an ongoing Phase 2 study, which showed that treatment with a combination of two cystic fibrosis transmembrane conductance regulator (CFTR) modulators, KALYDECO™ (ivacaftor, VX-770) and VX-809, may improve outcomes for people with the most common form of cystic fibrosis (CF) by targeting the underlying cause of the disease. |
| 2011-11-02 | Vertex Pharmaceuticals Incorporated | Results New England Journal of Medicine Publishes Data from Phase 3 STRIVE Study of KALYDECOTM (ivacaftor) in People Ages 12 and Older with a Specific Type of Cystic Fibrosis vrtx.com ↗
In this study, called STRIVE, people with CF ages 12 and older with at least one copy of the G551D mutation who were treated with KALYDECO (kuh-LYE-deh-koh) experienced significant improvements in lung function and other measures of disease. |
| 2011-10-19 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Submits Application for Priority Review and Approval of KALYDECO™ (VX-770, ivacaftor) in the U.S. as First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis vrtx.com ↗
The U.S. submission includes a request for Priority Review, which, if granted, would shorten the FDA's anticipated review time from 10 to six months. |
| 2011-06-10 | Vertex Pharmaceuticals Incorporated | Results Phase 3 STRIVE Study of VX-770 Showed Durable Improvements in Lung Function (FEV1) and Other Measures of Disease Among People With a Specific Type of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX - News) today announced the final results from its pivotal Phase 3 STRIVE study that evaluated VX-770, a medicine in development that targets the defective protein that causes cystic fibrosis (CF). |
| 2011-06-09 | Vertex Pharmaceuticals Incorporated | Results Interim Phase 2 Data Showed a Combination of VX-770 and VX-809 Improved Function of the Defective Protein that Causes Cystic Fibrosis in People With the Most Common Form of the Disease vrtx.com ↗ |
| 2011-03-29 | Vertex Pharmaceuticals Incorporated | Results Phase 3 Study of VX-770 in Children Ages 6 to 11 With a Specific Type of Cystic Fibrosis Showed Profound Improvements in Lung Function (FEV1) and Other Measures of Disease Through 24 Weeks vrtx.com ↗
A difference in mean absolute improvement from baseline in lung function of 12.5 percent and a difference in mean relative improvement from baseline in lung function of 17.4 percent compared to placebo (p0.0001) were observed through week 24. |
| 2011-02-23 | Vertex Pharmaceuticals Incorporated | Results Phase 3 Study of VX-770 Showed Profound and Sustained Improvements in Lung Function (FEV1) and Other Measures of Disease Among People With a Specific Type of Cystic Fibrosis vrtx.com ↗
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from the Phase 3 STRIVE study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). |
| 2010-11-17 | Vertex Pharmaceuticals Incorporated | Results New England Journal of Medicine Publishes Phase 2 Study of VX-770 as a New Approach to Treat the Underlying Cause of Cystic Fibrosis vrtx.com ↗
In a study published in this week's New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase 2 clinical trial of 39 people with cystic fibrosis (CF). |
| 2008-10-20 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive 28-Day Results for VX-770, an Oral Investigational Agent That Targets a Defective Protein Responsible for Cystic Fibrosis vrtx.com ↗
In this analysis, no patients discontinued treatment and no serious adverse events were reported. |
| 2008-03-27 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Positive Results for VX-770, an Oral Investigational Agent That Targets a Defective Protein Responsible for Cystic Fibrosis vrtx.com ↗
We saw an average improvement in lung function of 10 percent in patients receiving the highest dose, compared to no observed improvement in patients who received placebo, |
All press releases naming this drug 178 releases
| Date | Issuer | Release |
|---|---|---|
| Show all 178 releases opens the full record — a long page | ||
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 117 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ivacaftor | “received weight-based and age-based doses of oral lumacaftor-ivacaftor: children weighing less than 14 kg and aged younger than 6 years at study 116 screening received...” PMID 33965000 ↗ May 202198“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 ↗ “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 ↗ “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 ↗ “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 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 | IVA | “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 ↗26“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 ↗ “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 “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 ↗ “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 | IVA, VX-770 | “IVA, VX-770” NCT02392234 ↗ |
| Known as | Kalydeco | “This is a prospective randomized double-blinded placebo-controlled crossover study of the short-term effects of ivacaftor (aka VX-770 or Kalydeco) on sweat chloride...” NCT01784419 ↗4“The new drug, Ivacaftor (KALYDECO), is a drug that has recently been approved by the U.S. Food and Drug Administration (FDA) for patients with a lung disease called Cystic...” NCT03251911 ↗ “All participants in group 7 who received placebo matched to VX-661 tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days.” NCT01531673 ↗ |
| Known as | VX-770 | “This is a prospective randomized double-blinded placebo-controlled crossover study of the short-term effects of ivacaftor (aka VX-770 or Kalydeco) on sweat chloride...” NCT01784419 ↗17“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 “The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have...” NCT01262352 ↗ “Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning” NCT01931839 ↗ “lumacaftor (VX-809), a CFTR corrector, in combination with ivacaftor (VX-770), a CFTR potentiator” PMID 25981758 ↗ May 2015 “Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator” PMID 23590265 ↗ Jun 2013 “VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy” NCT02392234 ↗ “Ivacaftor (VX-770)” NCT03251911 ↗ “Ivacaftor (VX-770)” NCT07809867 ↗ |
| Action | Activate | “VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro.” PMID 21083385 ↗ Nov 2010 |
| Modality | Small molecule | “orally bioavailable small molecule” NCT01381289 ↗ |
| Route | Oral | “receive oral VX-770 every 12 hours” PMID 21083385 ↗ Nov 2010 |
| Target | CFTR | “Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein.” NCT00457821 ↗ |