Ivacaftor
Ivacaftor is a medication used to treat certain types of cystic fibrosis, a genetic disorder that affects the lungs and digestive system. It works by enhancing the function of a specific protein called CFTR, which is responsible for regulating salt and water movement in cells. This helps improve lung function and reduce respiratory symptoms in patients with specific mutations in the CFTR gene.
Approved by the U.S. Food and Drug Administration (FDA) in 2012, Ivacaftor is typically prescribed for patients aged 6 years and older who have at least one copy of the G551D mutation in the CFTR gene. It is taken orally, usually twice a day, and is often used in combination with other therapies to manage cystic fibrosis effectively.