CFTR modulator

Elexacaftor/tezacaftor/ivacaftor (Trikafta)

Your cells have tiny doors that let salt and water out. The CFTR gene is the blueprint for them, and cystic fibrosis is what happens when the blueprint has a typo. Trikafta puts the door back in place and opens it, but whether it works depends on which typo you carry.

What this drug connects to

The genes involved

CFTR

Cystic Fibrosis →

Trikafta (elexacaftor/tezacaftor/ivacaftor) is the medicine that reached the people ivacaftor could not. Think of CFTR as a door that lets salt and water out of cells. Ivacaftor can only open a door that is already in place; with F508del, the most common typo, the door never gets there. Trikafta adds two medicines that move the door into place, and then ivacaftor opens it. The US label, revised in 2026, covers people aged 2 and older with cystic fibrosis who have at least one CFTR variant that still makes some CFTR protein or that tests show responds to it; if the variants are not known, an FDA-cleared CF genetic test comes first. If the variants make no protein at all, there is no door to fix. Because it can seriously harm the liver, it carries a boxed warning and needs regular liver blood tests. Whether to take it is decided with a specialist, and nothing here is a reason to start, stop or change a medicine.

TRIKAFTA (elexacaftor, tezacaftor, and ivacaftor) US prescribing information, revised 03/2026 (Vertex Pharmaceuticals; FDA label 212273s014).

In the news

2026-04-01 · Vertex Pharmaceuticals (FDA label expansion covering any CFTR variant shown to still produce CFTR protein, not just a fixed list of named mutations)

Vertex Announces US FDA Approval for Label Extensions of ALYFTREK and TRIKAFTA, Expanding Availability to ~95% of All People With CF in the United States

FDA expanded the approved-use labels for two CFTR modulators -- Alyftrek (ages 6+) and Trikafta, which contains ivacaftor (ages 2+) -- from a fixed list of named mutations to any CFTR variant shown, by clinical or lab data, to still let the gene make CFTR protein: 564 variants qualify for Alyftrek, 521 for Trikafta. About 800 more people with CF in the US are now eligible, bringing total eligibility to roughly 95% of the US CF population -- a real-world example of exactly the variant-by-variant pharmacogenomic matching this page profiles.

Variants MyGeneLog™ reports for these genes

These are the positions in your own file that carry the genes above. Not every gene in a guideline is one we report — where that is the case the gene appears above without a variant here, and the note says so.

Barrett's esophagus or Esophageal adenocarcinoma

CFTR · rs17451754

Free Cholesterol to Cholesteryl Esters in Small HDL ratio

CFTR · rs145380885

Triglycerides to Total Lipids in Small HDL percentage

CFTR · rs6977665

Cystic fibrosis (F508del)

CFTR · rs113993960

Cystic fibrosis (G551D, a gating variant)

CFTR · rs75527207

Infective connective tissue disorders (PheCode 712)

CFTR · rs548335381

Questions about Elexacaftor/tezacaftor/ivacaftor (Trikafta)

What is Trikafta?

Trikafta is a cystic fibrosis medicine that puts three drugs in one: elexacaftor, tezacaftor and ivacaftor. Two of them straighten out the faulty CFTR "door" so it reaches its place on the cell, and the third opens it. Having cystic fibrosis is not enough to get it: what matters is which typo you carry in the CFTR gene.

Which CFTR variants qualify for Trikafta?

The US label covers anyone aged 2 or older with cystic fibrosis who has at least one CFTR variant that still makes some CFTR protein, even a misshapen one like F508del, or that tests have shown responds to the medicine. One such variant is enough. If your variants make no protein at all, there is no door for the medicine to fix.

Do I need a genetic test before Trikafta?

If your CFTR variants are not already known, yes. The label asks for an FDA-cleared CF genetic test first, to confirm you carry at least one variant the medicine can work on.

Is Trikafta the same as ivacaftor?

No, ivacaftor is one of its three parts. On its own, ivacaftor can only open a door that is already in place, as with G551D. With F508del, the most common typo, the door never reaches the cell surface, so ivacaftor alone has nothing to open. The two extra medicines in Trikafta move the door into place first.

Does Trikafta carry a safety warning?

Yes. Because it can seriously harm the liver, its label has a boxed warning, the FDA's strongest kind. So you have a liver blood test before starting, every month for the first 6 months, every 3 months for the next 12 months, and at least once a year after that. Whether to take it is decided with a cystic fibrosis specialist; nothing here is a reason to start, stop or change a medicine.

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Elexacaftor/tezacaftor/ivacaftor (Trikafta). MyGeneLog™. https://www.mygenelog.com/drugs/elexacaftor-tezacaftor-ivacaftor

This page is educational and contains no dosing information. Where a clinical guideline covers one of these gene-drug pairs it is written for prescribers and works through validated algorithms alongside clinical monitoring. Nothing here is a reason to start, stop, or change a medication — that conversation belongs with the clinician or pharmacist managing your treatment.