Olacaftor
As of the edition of 20 October 2026, Olacaftor is a small molecule: went quiet in 2017; acts on CFTR.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Went quiet, 2017
- Kind
- Small molecule
- Registered trials
- 2, 0 stopped early
- Disease areas
- Digestive system; Inherited and neonatal; Lungs and airways
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 1 | 1 | 1 | 0 | 0 |
| Phase 2 | 1 | 1 | 0 | 0 |
| All trials | 2 |
Who ran its trials
| Company | Role | Years |
|---|---|---|
| Vertex | Lead sponsor of trials | 2016 to now |