Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.
Lumacaftor is a CFTR corrector: it helps the F508del form of the CFTR protein fold properly and reach the cell surface. It is given only combined with ivacaftor, which then opens the channel, as Orkambi, for people with cystic fibrosis who carry two copies of F508del. Newer triple combinations work better for most patients.
Lumacaftor works only for cystic fibrosis caused by the F508del variant in CFTR, the gene behind the condition, and Orkambi is approved for people with two copies of it. All studies on this page concern CFTR variants. Lumacaftor strongly speeds up CYP3A4, which lowers the levels of ivacaftor and many other medicines. There is no CPIC or DPWG guideline for lumacaftor itself.
Every published finding on gene variants and Lumacaftor. Upload your DNA file to see which of these rows apply to you.
PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.
CFTR rs113993960: Level 3. Efficacy: Cystic Fibrosis. 3.
CFTR rs121909047: Level 3. Efficacy: Cystic Fibrosis. 1.
Studies of how well it worked in people carrying a variant.
CFTR rs113993960: Genotype del/del is associated with response to lumacaftor.
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.
More drugs from the same category
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.
Content updated September 2026.
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