The Medical Letter on Drugs and Therapeutics
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Ivacaftor (Kalydeco) for Cystic Fibrosis

The FDA has approved ivacaftor (eye va kaf’ tor; Kalydeco – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥6 years old with the G551D mutation, which is found in about 5% of patients with CF. It is the first drug approved in the US that treats the cause of the disease.

MECHANISM OF ACTION — The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel located in the apical membrane of epithelial cells responsible for transporting chloride and bicarbonate ions across the membrane. Mutations in the CFTR gene that encodes for this protein channel lead to formation of thick mucus buildup in the lungs and abnormal pancreatic and digestive tract function. Ivacaftor is a CFTR potentiator that improves chloride transport through CFTR channels with the G551D mutation.1 It is not effective in CF patients who are homozygous for the ... more      

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