Ivacaftor: A new emerging treatment option in the management of cystic fibrosis

Song, Jessica C.; Chiu, Hilda; Yoon, Jennefer
April 2012
Formulary;Apr2012, Vol. 47 Issue 4, p132
Academic Journal
Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been approved by FDA for treatment of cystic fibrosis in patients age 6 years and older who have the G551D mutation in the CFTR gene. This agent offers a novel approach to the treatment of cystic fibrosis, as it is the only marketed drug that targets the underlying cause of this lethal condition, instead of the downstream disease processes that occur as a result of lacking or defective CFTR. Available clinical trial data indicate that compared with placebo, ivacaftor has superior efficacy for improving respiratory function, and it has been shown to lower the risk of pulmonary exacerbations. The adverse-effect profile of ivacaftor does not differ substantially from placebo, but patients will need to be monitored for hepatic function and potential drug interactions during treatment.


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