Drugs / Lumacaftor
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed cystic fibrosis 2 milestones | |||||
| Filed | US (FDA) | ORKAMBI | cystic fibrosis | 2016-05-31 | “FDA has accepted for review a supplemental New Drug Application (sNDA) for the use of ORKAMBI® (lumacaftor/ivacaftor) in people with cystic fibrosis” vrtx.com ↗ “New Drug Application (NDA) submission of lumacaftor/ivacaftor in children ages 2 to 5 years old with a PDUFA date of August 7, 2018.” vrtx.com ↗ |
| Filed | EU (EMA) | ORKAMBI | cystic fibrosis | — | “an MAA line extension for lumacaftor/ivacaftor in this age group has been submitted to the EMA” vrtx.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Orkambi | — | 2015-11-19 | europa.eu ↗ |
| Approved | US (FDA) | ORKAMBI | — | 2015-07-02 | fda.gov ↗ |
Trials 28
| 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 | NCT01888393 | Jun → Nov 2013 | — | 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 | 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 · 2 missed | ||||||
| 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 | Missed primary |
| Phase 2 | NCT03061331 | Jan → Sep 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Completed | Missed primary |
| 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 · 4 met primary | ||||||
| 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 | Met primary |
| 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 | 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 42 trials · 1 missed | ||||||
| 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 | Missed primary |
| 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 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 |
| Comparator or background therapy2 trials | ||||||
| Phase 4 | NCT02823470 Background | Jun 2016 → Aug 2017 | cystic fibrosis | Vertex Pharmaceuticals Incorporated | Terminated | No outcome recorded Stop: Enrollment |
| — | 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 · 50
| Date | Issuer | Release |
|---|---|---|
| 2025-07-02 | Qanatpharma AG | Results QP Reports Topline Data from Phase 1 Trial in Healthy Volunteers qanatpharma.com ↗
Qanatpharma, a clinical-stage biopharma company specializing in microvascular research and medicine, announced publication of topline results from its Phase 1 bioequivalence and food effect trial that assessed the pharmacokinetics of Lumacaftor in 35 healthy adult volunteers (NCT05968612). |
| 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 |
| 2023-04-27 | 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 ↗ |
| 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. |
| 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. |
| 2019-10-21 | 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). |
| 2019-10-19 | Vertex Pharmaceuticals Incorporated | Regulatory 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 ↗
In addition, ORKAMBI® (lumacaftor/ivacaftor) is now also reimbursed for the treatment of children with CF ages 2 to 5 who have two copies of the F508del mutation in the CFTR gene. |
| 2019-06-04 | Vertex Pharmaceuticals Incorporated | Results Vertex to Present Data at ECFS Conference on Potential Impact of Early Treatment and Long-Term Treatment with CFTR Modulators on the Underlying Cause of CF vrtx.com ↗
The study showed a safety profile consistent with that observed in children aged 12 to <24 months and demonstrated substantial improvements in sweat chloride. |
| 2019-05-30 | Vertex Pharmaceuticals Incorporated | Results Vertex Selects Triple Combination Regimen of VX-445, Tezacaftor and Ivacaftor to Submit for Global Regulatory Approvals in Cystic Fibrosis vrtx.com ↗
Vertex previously announced that both of these Phase 3 studies met their primary endpoints, and the company today announced the final results of these studies, including results for key secondary endpoints and safety data. |
| 2019-03-12 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives Approval for SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) in Australia, to Treat the Underlying Cause of Cystic Fibrosis in People aged 12 and Older with Certain CFTR Gene Mutations vrtx.com ↗ |
| 2019-02-14 | Vertex Pharmaceuticals Incorporated | Results Positive Phase 3 Study for Tezacaftor/Ivacaftor Combination in Children Aged 6-11 Years with Cystic Fibrosis Supports European Medicines Agency Submission vrtx.com ↗
The study met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 8 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among patients treated with tezacaftor/ivacaftor. |
| 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. |
| 2018-11-16 | 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) |
| 2018-11-01 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene vrtx.com ↗
the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) |
| 2018-10-24 | Vertex Pharmaceuticals Incorporated | Results Vertex Reports Third-Quarter 2018 Financial Results vrtx.com ↗
the study's Data Safety Monitoring Board (DSMB) recommended to stop the study early due to futility. |
| 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. |
| 2018-07-27 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Receives European CHMP Positive Opinion for SYMKEVI® (tezacaftor/ivacaftor) for People with Cystic Fibrosis Aged 12 and Older with Certain Mutations in the CFTR Gene 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 SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. |
| 2018-06-07 | Vertex Pharmaceuticals Incorporated | Results Vertex Data Presented at European Cystic Fibrosis Society (ECFS) Conference Demonstrate Early and Long-Term Disease-Modifying Potential of Treating the Underlying Cause of CF vrtx.com ↗
Studies of investigational triple combination regimens highlight continued progress toward developing medicines to treat the underlying cause of the disease for up to 90 percent of people living with CF |
| 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. |
| 2017-11-10 | 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 |
| 2017-10-25 | Vertex Pharmaceuticals Incorporated | Results Vertex Reports Third-Quarter 2017 Financial Results vrtx.com ↗
In patients being treated with ORKAMBI, the addition of hypertonic saline resulted in a decrease in ppFEV1 of 0.1 percentage points at Day 28. |
| 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. |
| 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). |
| 2016-09-28 | 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. |
| 2016-06-10 | 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 ↗
Data from Phase 3 safety study of ORKAMBI in children ages 6-11 presented today at ECFS |
| 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. |
| 2016-01-26 | 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 ↗ |
| 2015-09-25 | 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 lumacaftor in combination with ivacaftor for people with cystic fibrosis ages 12 and older with two copies of the F508del 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 ↗ |
| 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 ↗ |
| 2014-10-09 | Vertex Pharmaceuticals Incorporated | Results 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 ↗
Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements in lung function through 48 weeks of treatment with lumacaftor in combination with ivacaftor in people with two copies of the F508del mutation |
| 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. |
| 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 |
| 2012-09-25 | 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. |
| 2012-06-28 | 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. |
| 2012-05-29 | 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) |
| 2012-05-07 | 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). |
| 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. |
| 2011-06-09 | 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 ↗ |
| 2010-02-03 | Vertex Pharmaceuticals Incorporated | Results Vertex Announces Results from Phase 2a Trial of VX-809 Targeting the Defective Protein Responsible for Cystic Fibrosis vrtx.com ↗
In the trial, VX-809 showed a statistically significant decline in sweat chloride at both the 100 mg and 200 mg once-daily doses, suggesting that the activity of the CFTR protein was increased in patients during dosing. |
All press releases naming this drug 63 releases
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” 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 |