Tezacaftor
As of the edition of 20 October 2026, Tezacaftor is a small molecule: marketed, first approved in 2018 (CA); acts on CFTR.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2024
- Kind
- Small molecule
- Brand name
- Alyftrek
- First approved
- 27 June 2018 (CA)
- Approvals on record
- 11 in 3 regions
- Holder of the most recent approval
- Vertex; 2 holders in all
- Registered trials
- 77, 3 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 | 11 | 1 | 5 | 0 |
| Phase 1 | 12 | 9 | 3 | 0 |
| Phase 2 | 11 | 11 | 0 | 0 |
| Phase 3 | 37 | 30 | 5 | 2 |
| Phase 4 | 6 | 2 | 3 | 1 |
| All trials | 77 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Vertex | Applicant and Lead sponsor of trials | 2018 to now |