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Ivacaftor therapy for cystic fibrosis

Introduction: Cystic fibrosis (CF) is a life-shortening autosomal recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF causes multisystem disease, most importantly pancreatic insufficiency and progressive bronchiectasis. Until recently, CF therapies have treated complications of end organ damage. Ivacaftor is the first commercially available therapy that improves CFTR function. In placebo-controlled clinical trials of CF subjects with the G551D and similar ‘gating’ CFTR mutations, ivacaftor-treated subjects showed improved pulmonary function, body weight and quality-of-life measures. Areas covered: This article discusses basic discoveries leading to the development of ivacaftor and results of Phase II and III clinical trials, accessed by a Medline search using key words ‘VX-770’ and ‘ivacaftor’; supplemental information was accessed using Google search and the US National Institutes of Health clinical trials website. Expert opinion: Ivacaftor is a breakthrough in CF therapy but is efficacious as a single agent in only a small percentage of CF patients. One study suggests that combining ivacaftor with the CFTR corrector therapy, lumacaftor, may benefit patients who are homozygous for the most common CFTR mutation, F508del. Importantly, the efficacy of ivacaftor demonstrates the potential of systemic, personalized therapy for CF.