Kalydeco Cystic Fibrosis
Kalydeco (ivacaftor) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Cystic fibrosis is caused by mutations in a gene that encodes for the CFTR protein that regulates ion (such as chloride) and water transport in the body. The defect in chloride and water transport results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems.Kalydeco was specifically approved for the treatment of cystic fibrosis in patients age 6 years and older who have a G551D mutation in the CFTR gene. If the patient?s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the G551D mutation.
Kalydeco is supplied as a tablet for oral administration. The recommended dose of Kalydeco for both adults and pediatric patients age 6 years and older is one 150 mg tablet taken orally every 12 hours (300 mg total daily dose).