elexacaftor/tezacaftor/ivacaftor
Trikafta (Kaftrio in Europe) combines elexacaftor, tezacaftor and ivacaftor from Vertex for cystic fibrosis. Two correctors help the defective CFTR channel reach the cell surface, and a potentiator opens it, restoring salt and water movement.
This page is based on FDA prescribing information, the EMA medicine record and Health Canada product information for elexacaftor/tezacaftor/ivacaftor, cross-checked with MedlinePlus; the mechanism is explained in plain language, and analogies are used only where they stay accurate.
How it works
Explains what the medicine does in the body, not dosing, side effects, or safety.
An analogy
In cystic fibrosis the CFTR channel is like a faulty gate that either never reaches the fence or arrives but will not open. Elexacaftor and tezacaftor are escorts that guide the gate into place, and ivacaftor is the key that turns in its lock. Once the gate works, salt and water flow again and mucus thins.
In simple terms
Cystic fibrosis is caused by mutations in the CFTR gene, which encodes a channel controlling salt and water movement on cell surfaces. When the channel fails, mucus becomes thick and sticky, especially in the lungs. This three-drug combination helps in two ways: elexacaftor and tezacaftor help the defective channel fold correctly and reach the cell surface, while ivacaftor helps the channel stay open. Together they improve CFTR function and reduce lung complications.
In depth
Elexacaftor and tezacaftor are CFTR correctors that bind distinct sites on the mutant CFTR protein, stabilising its folding and trafficking to the apical membrane, with additive effects in F508del homozygous and heterozygous genotypes. Ivacaftor is a potentiator that increases channel open probability once CFTR reaches the surface. The triple combination restores meaningful chloride transport in the large majority of people with CF, transforming a previously progressive disease.
See it in motion
A schematic animation of the mechanism — simplified for clarity, not drawn to scale.
The triple combination restores meaningful chloride transport in the large majority
- Cystic fibrosis is caused by mutations in the CFTR gene, which encodes a channel controlling salt and water movement on cell surfaces.
- In cystic fibrosis the CFTR channel is like a faulty gate that either never reaches the fence or arrives but will not open.
- This three-drug combination helps in two ways: elexacaftor and tezacaftor help the defective channel fold correctly and reach the cell surface, while ivacaftor helps the channel stay open.
- Once the gate works, salt and water flow again and mucus thins.
Target: CFTR chloride channel
A channel that moves chloride and water across cell surfaces; in cystic fibrosis CFTR mutations make the channel misfold or fail to open, producing thick mucus in the lungs and other organs.
Approved uses
Approved uses differ by country and change over time. Always follow your local prescribing information.
Approved uses in United States
FDA- Treatment of cystic fibrosis in adults and children 2 years of age and older who have at least one CFTR gene variant that is responsive based on clinical or in vitro data, or that results in residual CFTR function
Approved uses in European Union
EMA- In combination with ivacaftor, for cystic fibrosis in patients aged 6 years and older who have at least one non-Class I mutation in the CFTR gene
Official sources
The statements on this page trace back to these records.
- U.S. Food and Drug Administration: elexacaftor/tezacaftor/ivacaftor prescribing information
U.S. Food and Drug Administration
- MedlinePlus: elexacaftor, tezacaftor, and ivacaftor
MedlinePlus, U.S. National Library of Medicine
This page explains how the medicine works — it isn’t the patient leaflet. For anything that applies to you, read the leaflet that came with your medicine or ask your pharmacist.
MedUnfold provides educational content and is not medical advice. Always follow your local prescribing information and healthcare professional.