elexacaftor
Trade name: Trifakta
Small moleculeapprovedOrphan Drug FDAFast Track FDABreakthrough Therapy FDA
Approved
Oct 21, 2019
Clinical trial activity
29 trials · 14 clinical orgs · 2 marketing orgs
Phase 1
1
Phase 2
24
Phase 3
23
Phase 4
3
Earliest trial started Jan 23, 2017 (NCT03227471)
Timeline
Indications
Approved for
Mechanism of action
CFTR activator
- Cystic fibrosis transmembrane conductance regulatorPOSITIVE MODULATOR
Cystic fibrosis transmembrane conductance regulator positive modulator
Facilitates processing and trafficking of CFTR mutants to increase the amount of mature protein at the cell surface. Has an additive effect with IVACAFTOR AND TEZACAFTOR on functional CFTR.
Combination products
trikafta (copackaged)
with ivacaftor, tezacaftor
Also known as elexacaftor and tezacaftor and ivacaftor, elexacaftor/ivacaftor/tezacaftor, elexacaftor/tezacaftor/ivacaftor, kaftrio
Approval history
- approvedPriority reviewFast trackBreakthroughOct 21, 2019
Chemistry & pharmacology
Loading structure…
SMILES
Cc1nn(C)cc1S(=O)(=O)NC(=O)c1ccc(-n2ccc(OCC(C)(C)C(F)(F)F)n2)nc1N1C[C@@H](C)CC1(C)C- Mol. weight
- 597.66 g/mol
- Lipinski Ro5
- 1 violation(s)
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Sources
- NCATSRRN67GMB0V ↗
- ChEMBLCHEMBL4298128 ↗
Also known as
- vx-445
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06460506 | Phase 3 | Nov 21, 2024 | Vertex |
| NCT05882357 | Phase 3 | Jun 27, 2023 | Vertex |
| NCT05743946 | Phase 4 | Apr 18, 2023 | Emory University, The Marcus Foundation |
| NCT05331183 | Phase 3 | Nov 23, 2022 | Vertex |
| NCT05274269 | Phase 3 | May 9, 2022 | Vertex |
| NCT05153317 | Phase 3 | Jan 31, 2022 | Vertex |
| NCT05111145 | Phase 3 | Jan 31, 2022 | Vertex |
| NCT05076149 | Phase 3 | Oct 27, 2021 | Vertex |
| NCT04969224 | Phase 3 | Oct 12, 2021 | Vertex |
| NCT04840862 | Phase 4 | Sep 30, 2021 | University of Southern California |
| NCT05033080 | Phase 3 | Sep 14, 2021 | Vertex |
| NCT04545515 | Phase 3 | Jan 31, 2021 | Vertex |
| NCT04599465 | Phase 3 | Jan 18, 2021 | Vertex |
| NCT04537793 | Phase 3 | Nov 19, 2020 | Vertex |
| NCT04602468 | Phase 4 | Sep 3, 2020 | Cystic Fibrosis Registry of Ireland, Erasmus University Rotterdam, Imperial College London, Medizinische Hochschule Brandenburg Theodor Fontane, National University of Ireland, Queen's University, Belfast, Royal College of Surgeons, Ireland, Teagasc, University of Limerick, University of Toronto |
| NCT04353817 | Phase 3 | Jun 30, 2020 | Vertex |
| NCT04362761 | Phase 3 | Apr 30, 2020 | Vertex |
| NCT04183790 | Phase 3 | Feb 29, 2020 | Vertex |
| NCT04058366 | Phase 3 | Nov 30, 2019 | Vertex |
| NCT04105972 | Phase 3 | Sep 30, 2019 | Vertex |
Showing 20 of 29 trials
Page 1 / 2
Organizations
Research & Development (14)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Vertex | For profit | 26 | 26 | 3 | 2017 |
| Cystic Fibrosis Registry of Ireland | Academic/Hospital | 1 | 0 | 1 | 2020 |
| Emory University | Academic/Hospital | 1 | 1 | 1 | 2023 |
| Erasmus University Rotterdam | Academic/Hospital | 1 | 0 | 1 | 2020 |
| Imperial College London | Academic/Hospital | 1 | 0 | 1 | 2020 |
| Medizinische Hochschule Brandenburg Theodor Fontane | Academic/Hospital | 1 | 0 | 1 | 2020 |
| National University of Ireland | Academic/Hospital | 1 | 0 | 1 | 2020 |
| Queen's University, Belfast | Academic/Hospital | 1 | 0 | 1 | 2020 |
| Royal College of Surgeons, Ireland | Academic/Hospital | 1 | 1 | 1 | 2020 |
| Teagasc | Government | 1 | 0 | 1 | 2020 |
| The Marcus Foundation | Academic/Hospital | 1 | 0 | 1 | 2023 |
| University of Limerick | Academic/Hospital | 1 | 0 | 1 | 2020 |
| University of Southern California | Academic/Hospital | 1 | 1 | 1 | 2021 |
| University of Toronto | Academic/Hospital | 1 | 0 | 1 | 2020 |
14 organizations
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