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Tezacaftor/Ivacaftor
Tezacaftor is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly, while ivacaftor is a CFTR potentiator that increases the channel-opening probability of CFTR at the cell surface.
Tezacaftor is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly, while ivacaftor is a CFTR potentiator that increases the channel-opening probability of CFTR at the cell surface. Used for Cystic fibrosis in patients aged 6 years and older with at least one F508del mutation, Cystic fibrosis in patients with other CFTR mutations responsive to CFTR modulation.
At a glance
| Generic name | Tezacaftor/Ivacaftor |
|---|---|
| Also known as | TEZ/IVA; VX-661/VX-770, VX-661/VX-770, TEZ/IVA |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | CFTR modulator (corrector + potentiator combination) |
| Target | CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) |
| Modality | Small molecule |
| Therapeutic area | Pulmonary/Respiratory |
| Phase | Phase 3 |
Mechanism of action
Tezacaftor addresses the trafficking defect of F508del-CFTR and other Class II mutations by promoting proper protein folding in the endoplasmic reticulum, allowing more CFTR to reach the cell membrane. Ivacaftor then enhances the function of CFTR channels that are present at the cell surface by increasing their open probability, improving chloride transport. Together, this combination addresses both the quantity and function of CFTR protein.
Approved indications
- Cystic fibrosis in patients aged 6 years and older with at least one F508del mutation
- Cystic fibrosis in patients with other CFTR mutations responsive to CFTR modulation
Common side effects
- Headache
- Nausea
- Diarrhea
- Abdominal pain
- Rash
- Elevated liver transaminases
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)
- Personalized Theratyping Trial (EARLY_PHASE1)
- 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)
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:
- Tezacaftor/Ivacaftor CI brief — competitive landscape report
- Tezacaftor/Ivacaftor updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI