Trikafta

Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.

About Trikafta

What it is

Elexacaftor/tezacaftor/ivacaftor is a triple CFTR modulator for cystic fibrosis. Elexacaftor and tezacaftor help the faulty CFTR protein fold and reach the cell surface, and ivacaftor holds its channel open, so chloride and water move again and mucus becomes thinner. It has transformed treatment for most people with cystic fibrosis. It is sold as Trikafta and, in Europe, Kaftrio (taken with ivacaftor).

How genes come into it

Whether it works depends on the CFTR variants that cause a person's cystic fibrosis. It is approved for people with at least one F508del copy, the most common cause, and for many other CFTR variants shown to respond, which the US label lists. All studies on this page concern CFTR variants. It is broken down by CYP3A4, so medicines that block or speed up that enzyme change its levels.

What research found

Every published finding on gene variants and Trikafta. Upload your DNA file to see which of these rows apply to you.

Rated evidence (16 rated variants)

PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.

CFTR rs186089140: Level 1A · replicated. Efficacy: Cystic Fibrosis. 2.

CFTR rs36210737: Level 1A · replicated. Efficacy: Cystic Fibrosis. 2.

CFTR rs77932196: Level 1A · replicated. Efficacy: Cystic Fibrosis. 2.

CFTR rs121908751: Level 1A · replicated. Efficacy: Cystic Fibrosis. 1.

Response (26 findings)

Studies of how well it worked in people carrying a variant.

CFTR rs75389940: Genotypes AG + GG is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor or ivacaftor / tezacaftor in people with Cystic Fibrosis.

CFTR rs80034486: Allele G is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor in people with Cystic Fibrosis as compared to allele C.

CFTR rs121909011: Allele T is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor in people with Cystic Fibrosis.

CFTR rs121909019: Allele T is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor in people with Cystic Fibrosis.

Other findings (3 findings)

Studies that do not fall into the groups above.

CFTR CFTR 3849+10kbC->T: CFTR 3849+10kbC->T is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor in people with Cystic Fibrosis.

CFTR rs758900656: Allele A is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor.

CFTR rs186089140: Allele T is associated with increased clinical benefit to elexacaftor / tezacaftor / ivacaftor.

Study findings and evidence levels: PharmGKB (CC BY-SA 4.0). Guidelines: CPIC and DPWG, as bundled in PharmCAT. Most studies were done mainly in people of European ancestry.

Related Drugs

More drugs from the same category

Kalydeco (CFTR Modulators)

Lumacaftor (CFTR Modulators)

Orkambi (CFTR Modulators)

Not a prescribing recommendation. This page is educational information about pharmacogenomic research and clinical guidelines - it is not medical or dosing advice, and not a prediction of how any medication will work for you. Never start, stop, or change a medication or dose based on it; talk to your doctor or pharmacist, who can order clinical-grade testing if appropriate.

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Content updated September 2026.

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