Small molecule · Modulator of CFTR · developed by Vertex Pharmaceuticals
Elexacaftor is a small-molecule CFTR modulator that binds to CFTR protein and facilitates its cellular processing and trafficking to the cell surface. Combined with tezacaftor and ivacaftor in TRIKAFTA, it increases CFTR quantity and function, enhancing chloride transport in patients with cystic fibrosis who carry responsive CFTR variants.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Jun 22, 2026). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT06460506 | Phase 3 | Cystic Fibrosis | Active not recruiting | VRTX |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Cystic Fibrosis
Updated Oct 3, 2026. Something wrong or missing? Report it on the VRTX page.
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