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Elexacaftor / Ivacaftor / Tezacaftor
This triple combination corrects defective cystic fibrosis transmembrane conductance regulator (CFTR) protein by enhancing its production, localization, and function.
This triple combination corrects defective cystic fibrosis transmembrane conductance regulator (CFTR) protein by enhancing its production, localization, and function. Used for Cystic fibrosis in patients aged 6 years and older with at least one F508del-CFTR mutation or other eligible CFTR mutations.
At a glance
| Generic name | Elexacaftor / Ivacaftor / Tezacaftor |
|---|---|
| Also known as | Kaftrio, Trikafta, ETI, Kaftrio®, Triple Combination CFTR Modulator Therapy, ETI - Elexacaftor/Tezacaftor/Ivacaftor |
| Sponsor | Nottingham University Hospitals NHS Trust |
| Drug class | CFTR modulator combination (correctors + potentiator) |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Respiratory / Genetic |
| Phase | FDA-approved |
Mechanism of action
Elexacaftor and tezacaftor are CFTR correctors that help the mutant CFTR protein fold properly and traffic to the cell surface, while ivacaftor is a CFTR potentiator that increases the channel's open probability once at the membrane. Together, they restore ion and fluid transport in the airways and other organs affected by cystic fibrosis.
Approved indications
- Cystic fibrosis in patients aged 6 years and older with at least one F508del-CFTR mutation or other eligible CFTR mutations
Common side effects
- Headache
- Nausea
- Diarrhea
- Rash
- Elevated transaminases
- Abdominal pain
Key clinical trials
- Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older (PHASE3)
- 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)
- Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis.
- Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del (PHASE3)
- Glucose Metabolism in Cystic Fibrosis Related Diabetes (CFRD)
- Prevalence of Exercise-induced Ventilatory Limitation and Associated Factors in Patients With Cystic Fibrosis Receiving Elexacaftor-Tezacaftor-Ivacaftor (NA)
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
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