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Ivacaftor · Kalydeco, IVA

Small molecule · Acts on CFTR · developed by Vertex Pharms

Approved FDA, Jan 2012

VRTX Developer

1 Registered trials

What is Ivacaftor?

Ivacaftor is a small-molecule potentiator of the CFTR chloride channel protein. It increases chloride transport by enhancing the channel open probability of CFTR protein at the cell surface in patients with responsive CFTR mutations.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Kalydeco, IVA
Drug type: Small molecule
Targets: CFTR
Mechanism: Acts on CFTR
Pathways: chloride transport, CFTR gating
Therapy area: Respiratory
Developer: Vertex Pharms (VRTX)
FDA status: Approved as Kalydeco (January 31, 2012), NDA203188 · FDA label

✅ FDA-approved uses

Cystic fibrosis

From the indications section of the FDA label (version of Mar 23, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

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.

Conditions studied

Cystic Fibrosis

Other drugs acting on CFTR

Deutivacaftor FDA approved Elexacaftor FDA approved Tezacaftor FDA approved Vanzacaftor FDA approved

More from Vertex Pharms

Deutivacaftor FDA approved Elexacaftor FDA approved Exagamglogene Autotemcel FDA approved Suzetrigine FDA approved Tezacaftor FDA approved Vanzacaftor FDA approved Inaxaplin Phase 2/3 Povetacicept Phase 2/3 VX-017 Phase 3 VX-118 Phase 1 VX-264 Phase 1/2 VX-272 Phase 1

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the VRTX page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z