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VX-770
VX-770 is a CFTR potentiator that increases the opening probability and duration of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel at the cell surface.
VX-770 is a CFTR potentiator that increases the opening probability and duration of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel at the cell surface. Used for Cystic fibrosis with G551D-CFTR mutation, Cystic fibrosis with other gating mutations.
At a glance
| Generic name | VX-770 |
|---|---|
| Also known as | ivacaftor, Ivacaftor |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CFTR potentiator |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Respiratory / Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
VX-770 binds to CFTR and enhances its function by increasing the time the channel spends in the open state, allowing greater chloride ion transport across epithelial cell membranes. This mechanism is particularly effective for cystic fibrosis patients with gating mutations (such as G551D) where the CFTR protein is present but dysfunctional. By restoring chloride channel function, VX-770 reduces the viscosity of airway secretions and improves lung function.
Approved indications
- Cystic fibrosis with G551D-CFTR mutation
- Cystic fibrosis with other gating mutations
Common side effects
- Headache
- Upper respiratory tract infection
- Oropharyngeal pain
- Diarrhea
- Nausea
Key clinical trials
- Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation
- A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) (PHASE1, PHASE2)
- Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. (NA)
- Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older (PHASE3)
- Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants (PHASE2)
- Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
- Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
- Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- VX-770 CI brief — competitive landscape report
- VX-770 updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI