Elexacaftor
As of the edition of 20 October 2026, Elexacaftor is a small molecule: marketed, first approved in 2020 (EU); acts on CFTR.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2021
- Kind
- Small molecule
- Brand name
- Kaftrio
- First approved
- 21 August 2020 (EU)
- Approvals on record
- 5 in 2 regions
- Holder of the most recent approval
- Vertex; 2 holders in all
- Registered trials
- 38, 0 stopped early
- Disease areas
- Digestive system; Inherited and neonatal; Lungs and airways; Nervous system
US protection
No US approval on record.
With no US approval on record there are no listed US patents or exclusivities.
This is the US only.
Target
| Gene | Protein | UniProt |
|---|---|---|
| CFTR | Cystic fibrosis transmembrane conductance regulator | P13569 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 10 | 1 | 5 | 0 |
| Phase 2 | 2 | 2 | 0 | 0 |
| Phase 3 | 21 | 19 | 2 | 0 |
| Phase 4 | 5 | 2 | 3 | 0 |
| All trials | 38 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Vertex | Applicant and Lead sponsor of trials | 2020 to now |