Lumacaftor
As of the edition of 20 October 2026, Lumacaftor is a small molecule: marketed, first approved in 2015 (US); acts on CFTR; no US protection is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2016
- Kind
- Small molecule
- Brand name
- Orkambi
- First approved
- 2 July 2015 (US)
- Approvals on record
- 11 in 3 regions
- Holder of the most recent approval
- Vertex; 2 holders in all
- Registered trials
- 31, 4 stopped early
- Disease areas
- Inherited and neonatal; Digestive system; Lungs and airways; Nutrition and metabolism; Hormones; Heart and blood vessels
US protection
No US protection is listed.
The FDA registers list no drug-substance patent or exclusivity that counts for this drug.
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 | 7 | 5 | 0 | 2 |
| Phase 1 | 7 | 7 | 0 | 0 |
| Phase 2 | 7 | 6 | 1 | 0 |
| Phase 3 | 7 | 7 | 0 | 0 |
| Phase 4 | 3 | 1 | 0 | 2 |
| All trials | 31 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Vertex | Applicant and Lead sponsor of trials | 2009 to now |