Drugs / Lumacaftor
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Orkambi | — | 2015-11-19 | europa.eu ↗ |
| Approved | US (FDA) | ORKAMBI | — | 2015-07-02 | fda.gov ↗ |
Trials 28 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 16 trials | ||||||
| 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 | NCT00966602 | Sep → Dec 2009 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 26 trials | ||||||
| Phase 2 | NCT07695090 | Jul 2026 → May 2027 expected | symptomatic heart failure | Qanatpharma AG | Recruiting | No outcome recorded |
| Phase 2 | NCT04581408 | Jun 2021 → Apr 2025 | long QT syndrome | Istituto Auxologico Italiano | Completed | No outcome recorded |
| Phase 2 | NCT03625466 | Aug 2018 → Oct 2020 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT03061331 | Jan → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT01225211 | Oct 2010 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 2 | NCT00865904 | Mar → Dec 2009 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 310 trials | ||||||
| Phase 3 | NCT04235140 | Feb 2020 → Aug 2023 | 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 | NCT02797132 | May 2016 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02544451 | Aug 2015 → Aug 2018 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02514473 | Jul 2015 → Sep 2016 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT02390219 | Mar 2015 → Oct 2016 | cystic fibrosis, lung disorder | 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 | No outcome recorded |
| Phase 3 | NCT01807923 | May 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 3 | NCT01807949 | Apr 2013 → Apr 2014 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase 42 trials | ||||||
| Phase 4 | NCT03956589 | Oct 2018 → Apr 2020 | cystic fibrosis | University Hospital, Antwerp | Terminated | No outcome recorded |
| Phase 4 | NCT02875366 | Sep 2016 → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | No outcome recorded |
| Phase not applicable4 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 |
| — | 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 |
Also used as a comparator or background therapy in 2 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 41 trial | ||||||
| Phase 4 | NCT02823470 Background | Jun 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT03894657 Background | Dec 2019 → Nov 2021 | cystic fibrosis | Assistance Publique - Hôpitaux de Paris | Completed | No outcome recorded |
Press releases naming this drug 47 releases
| Date | Issuer | Release |
|---|---|---|
| 2023-07-05 | Vertex Pharmaceuticals Incorporated | European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old vrtx.com ↗ |
| 2023-04-27 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old vrtx.com ↗ |
| 2022-09-02 | Vertex Pharmaceuticals Incorporated | Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months vrtx.com ↗ |
| 2020-04-21 | Vertex Pharmaceuticals Incorporated | Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI® (lumacaftor/ivacaftor) and SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Cystic Fibrosis Patients vrtx.com ↗ |
| 2019-11-20 | Vertex Pharmaceuticals Incorporated | French Authorities Approve National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) for Eligible People Ages Two and Older With Cystic Fibrosis vrtx.com ↗ |
| 2019-10-21 | Vertex Pharmaceuticals Incorporated | Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor) vrtx.com ↗ |
| 2019-10-19 | Vertex Pharmaceuticals Incorporated | Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and ORKAMBI® (lumacaftor/ivacaftor) in Children Ages 2 to 5, With Certain CFTR Mutations in Australia vrtx.com ↗ |
| 2019-09-12 | Vertex Pharmaceuticals Incorporated | Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) vrtx.com ↗ |
| 2019-01-21 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2018-12-19 | Vertex Pharmaceuticals Incorporated | Vertex Announces it will Submit Cystic Fibrosis Medicines ORKAMBI® (lumacaftor/ivacaftor) as well as SYMKEVI® (tezacaftor/ivacaftor) to be Used in Combination with ivacaftor, to the Scottish Medicines Consortium for Appraisal vrtx.com ↗ |
| 2018-12-13 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2018-11-16 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2018-09-03 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2018-08-20 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2018-08-07 | Vertex Pharmaceuticals Incorporated | FDA Approves ORKAMBI® (lumacaftor/ivacaftor) as First Medicine to Treat the Underlying Cause of Cystic Fibrosis for Children Ages 2-5 Years with Most Common Form of the Disease vrtx.com ↗ |
| 2018-06-18 | Vertex Pharmaceuticals Incorporated | Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor) vrtx.com ↗ |
| 2018-01-10 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2017-11-10 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2017-07-13 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2017-06-09 | Vertex Pharmaceuticals Incorporated | Vertex Announces Nine Presentations of Data on ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) at the European Cystic Fibrosis Society (ECFS) Conference vrtx.com ↗ |
| 2017-06-01 | Vertex Pharmaceuticals Incorporated | Vertex Announces Long-Term Reimbursement Agreement with the Republic of Ireland for ORKAMBI® (lumacaftor/ivacaftor), KALYDECO® (ivacaftor) and Future Cystic Fibrosis Medicines vrtx.com ↗ |
| 2016-12-19 | Vertex Pharmaceuticals Incorporated | Vertex Announces German Reimbursement Agreement 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 ↗ |
| 2016-10-27 | Vertex Pharmaceuticals Incorporated | Vertex Presents Long-Term Data Demonstrating that ORKAMBI® (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) Show the Potential to Modify the Progression of CF vrtx.com ↗ |
| 2016-09-28 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2016-06-10 | Vertex Pharmaceuticals Incorporated | Vertex Announces Presentations of Data for KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) at European Cystic Fibrosis Society (ECFS) Conference vrtx.com ↗ |
| 2016-05-31 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2016-03-08 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2016-01-26 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2015-11-20 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2015-09-25 | Vertex Pharmaceuticals Incorporated | Vertex Receives CHMP Positive Opinions for ORKAMBI™ (lumacaftor/ivacaftor) and KALYDECO® (ivacaftor) in the European Union vrtx.com ↗ |
| 2015-07-02 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2015-07-02 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2015-05-17 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2015-05-12 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2014-11-05 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2014-06-24 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2013-02-26 | Vertex Pharmaceuticals Incorporated | Vertex Announces Initiation of Pivotal Phase 3 Program of VX-809 in Combination with Ivacaftor for the Treatment of People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗ |
| 2012-10-11 | Vertex Pharmaceuticals Incorporated | Data from Phase 2 Combination Study of VX-809 and Ivacaftor in People with Cystic Fibrosis Who Have the Most Common Genetic Mutation (F508del) Presented at North American Cystic Fibrosis Conference vrtx.com ↗ |
| 2012-06-28 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2012-05-29 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2012-05-07 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2011-11-03 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2011-10-03 | Vertex Pharmaceuticals Incorporated | Vertex Announces Presentation of New Data on VX-770 and VX-809 at North American Cystic Fibrosis Conference vrtx.com ↗ |
| 2011-06-09 | Vertex Pharmaceuticals Incorporated | 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 ↗ |
| 2010-10-18 | Vertex Pharmaceuticals Incorporated | Vertex Pharmaceuticals Initiates Clinical Trial to Evaluate Combination Regimens of VX-770 and VX-809 Targeting the Defective Protein Responsible for Cystic Fibrosis vrtx.com ↗ |
| 2010-02-03 | Vertex Pharmaceuticals Incorporated | Vertex Announces Results from Phase 2a Trial of VX-809 Targeting the Defective Protein Responsible for Cystic Fibrosis vrtx.com ↗ |
| 2009-03-25 | Vertex Pharmaceuticals Incorporated | Vertex Pharmaceuticals Initiates Phase 2 Development for CFTR Corrector VX-809 in Patients with Cystic Fibrosis vrtx.com ↗ |
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 33 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | lumacaftor | “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 202125“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 ↗ “Over 120 weeks (initial study period and follow-up), systolic blood pressure rose by 5.1 mmHg and diastolic blood pressure by 4.1 mmHg with twice-daily 400 mg...” PMID 37983082 ↗ Nov 2023 “Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial.” PMID 39173175 ↗ Nov 2024 “Orkambi® is a combination of Ivacaftor (a CFTR channel potentiator) and Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR).” NCT03894657 ↗ “Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).” NCT02544451 ↗ “Standard of care: Lumacaftor with Ivacaftor or Tezacaftor combined with Ivacaftor.” NCT04415268 ↗ “Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation.” PMID 33249003 ↗ Nov 2020 “Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function” NCT03956589 ↗ “Children received lumacaftor 100 mg and ivacaftor 125 mg” PMID 30686767 ↗ Jan 2019 “lumacaftor/ivacaftor” NCT02823470 ↗ “Lumacaftor/Ivacaftor” NCT02875366 ↗ |
| Known as | LUM | “Lumacaftor (LUM) is a drug developed and currently indicated for the treatment of cystic fibrosis (CF) in patients homozygous for the F508del mutation in the CFTR gene.” NCT04581408 ↗2“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 (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).” NCT02544451 ↗ |
| Known as | Lumacaftor component of orkambi | ChEMBL registry synonym — accepted as the source's own label CHEMBL2103870 ↗ |
| Known as | VRT 826809 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2103870 ↗ |
| Known as | VX-809 | “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 ↗5“lumacaftor (VX-809), a CFTR corrector” PMID 25981758 ↗ May 2015 |
| Action | Restore | “Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR)” NCT03894657 ↗ |
| Modality | Small molecule | “Lumacaftor is an existing small-molecule CFTR-corrector” NCT07695090 ↗ |
| Route | Oral | “Pharmacokinetic data supported a once-daily oral dosing regimen.” PMID 21825083 ↗ Aug 2011 |
| Target | CFTR | “VX-809, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, has been shown to increase the cell surface density of functional F508del-CFTR in vitro.” PMID 21825083 ↗ Aug 2011 |