Ivacaftor: a guide to its use in cystic fibrosis

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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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