Abstract
Ivacaftor (Kalydeco®) is the first drug approved for the treatment of cystic fibrosis that treats the underlying cause of the disease. It potentiates the open probability (i.e. gating) of cystic fibrosis transmembrane conductance regulator (CFTR) channels with a G551D, G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N or S549R gating mutation, thus enhancing their transport of chloride. When combined with standard care, ivacaftor significantly improved outcomes related to lung function, body mass index, pulmonary exacerbations and patient-reported respiratory symptoms relative to placebo in phase 3 trials in patients aged ≥6 years carrying a G551D or a specific non-G55ID mutation in the CFTR gene. The drug was generally well tolerated for up to 144 weeks.
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Acknowledgments
The manuscript was reviewed by: F. Becq, Institut de Physiologie et Biologie Cellulaires, Université de Poitiers, Poitiers, France; S. Saluja, Consultant in Internal Medicine, Saran Ashram, Agra, India.
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This article was updated from Drugs 2013; 73(14):1595–1604 [2] by contracted/salaried employees of Adis/Springer. The preparation of this review was not supported by any external funding. During the peer review process, the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from comments received were made by the authors on the basis of scientific and editorial merit.
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Lyseng-Williamson, K.A., Yang, L.P.H. & Deeks, E.D. Ivacaftor: a guide to its use in cystic fibrosis. Drugs Ther Perspect 31, 17–22 (2015). https://doi.org/10.1007/s40267-014-0178-5
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DOI: https://doi.org/10.1007/s40267-014-0178-5