Cystic fibrosis (CF) is a genetic disease associated with specific gene mutations that presents with pulmonary inflammation and frequent lung infections, exocrine pancreatic insufficiency, altered sweat composition and declining lung function. Ivacaftor (Kalydeco®) was approved for treatment of cystic fibrosis in patients 6 years of age and older with a G551D mutation on the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a CFTR potentiator and does not work in patients with a mutation of the F508del. Efficacy has been demonstrated in several trials with a primary outcome of improved FEV1, improvements in pulmonary exacerbations, patient-reported decrease in respiratory symptoms and weight gain. Side effects that have been reported include oropharyngeal pain, nasal congestion, abdominal pain, upper respiratory tract infection, rash and dizziness. The drug is metabolized via the CYP3A4 enzyme system and should be monitored for potential drug interactions accordingly. Information on long-term safety is not yet available, but clearly this drug represents an advance in the management of a debilitating disease.
Skouby A, Kintner K, Loughlin K, Blum E, Rush M. Overview of Kalydeco® (Ivacaftor) for Treatment of Cystic Fibrosis. PAW Review. 2014 Feb 01; 5(1):Article 6 32-37 . Available from: https://digitalcommons.onu.edu/paw_review/vol5/iss1/6. Free full text article.