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Information about Ivacaftor

Ivacaftor, lumacaftor and tezacaftor are orally available potentiators or correctors of the cystic fibrosis transmembrane conductance regulator (CFTR) that are used to treat patients with cystic fibrosis with specific mutations of the CFTR. 

Liver safety of Ivacaftor

Ivacaftor alone or in combination with lumacaftor or tezacaftor has been associated with transient serum enzyme elevations during treatment, but neither agent has been convincingly implicated in cases of clinically apparent acute liver injury with jaundice.

Mechanism of action of Ivacaftor

Ivacaftor (eye" va kaf' tor) was the first CFTR modulator to become available for use in the United States.  It potentiates the opening of the CFTR channel in patients who harbor at least one mutation in the CFTR gene that is responsive to invacaftor based upon clinical or in vitro assay data, such as the Gly551Asp (also abbreviated as G551D) CFTR mutation.  Ivacaftor was approved in the United States for use in patients with the CFTR Gly551Asp mutation in 2012 and is available as monotherapy in tablets of 150 mg and as oral granules in packets of 50 and 75 mg under the brand name Kalydeco.  The recommended dose in adults and children above 6 years of age is 150 mg orally every 12 hours.  The dose in children less than 6 years of age is based upon body weight.  The Gly551Asp mutation is found in approximately 5% of patients with CF.

genetic disorder agents

cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents


homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

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