Ivacaftor is a medication used to treat cystic fibrosis (CF) in patients who have specific genetic mutations. It works by targeting the defective protein that causes CF, helping to improve lung function, reduce exacerbations, and improve quality of life for patients with the disease. Research in this area focuses on studying the efficacy and safety of ivacaftor in different patient populations, as well as exploring potential combination therapies to further improve outcomes for individuals with CF. Additionally, researchers are investigating the long-term effects of ivacaftor treatment and its role in potentially modifying the progression of the disease.