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Ivacaftor (VX-770)
Ivacaftor is a CFTR potentiator that increases the open probability and activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein at the cell surface.
Ivacaftor is a CFTR potentiator that increases the open probability and activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein at the cell surface. Used for Cystic fibrosis in patients with G551D mutation in CFTR gene, Cystic fibrosis in patients with other gating mutations (R117H, R334W, R347P, R352Q, A455E, S549N, S549R, G1244E, S1251N, S1255P, S1255R, G1349D), Cystic fibrosis in patients with F508del-CFTR mutation (in combination with lumacaftor or tezacaftor).
At a glance
| Generic name | Ivacaftor (VX-770) |
|---|---|
| Sponsor | University of Alabama at Birmingham |
| Drug class | CFTR potentiator |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Respiratory / Genetic Disorders |
| Phase | FDA-approved |
Mechanism of action
Ivacaftor binds directly to the CFTR protein and enhances its channel-opening activity, allowing increased chloride ion transport across epithelial cell membranes. This mechanism is particularly effective for CFTR mutations that produce protein that reaches the cell surface but has reduced function (gating mutations). By restoring CFTR function, ivacaftor improves mucociliary clearance and reduces airway inflammation in cystic fibrosis patients.
Approved indications
- Cystic fibrosis in patients with G551D mutation in CFTR gene
- Cystic fibrosis in patients with other gating mutations (R117H, R334W, R347P, R352Q, A455E, S549N, S549R, G1244E, S1251N, S1255P, S1255R, G1349D)
- Cystic fibrosis in patients with F508del-CFTR mutation (in combination with lumacaftor or tezacaftor)
Common side effects
- Headache
- Oropharyngeal pain
- Upper respiratory tract infection
- Nausea
- Diarrhea
- Rash
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:
- Ivacaftor (VX-770) CI brief — competitive landscape report
- Ivacaftor (VX-770) updates RSS · CI watch RSS
- University of Alabama at Birmingham portfolio CI