ivacaftor

Ivacaftor is a small molecule pharmaceutical drug used to treat cystic fibrosis (CF) in patients with specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, such as G551D and others. It is the first medication to target the underlying cause of CF rather than just its symptoms. Ivacaftor acts as a CFTR potentiator, increasing the probability that defective CFTR chloride channels remain open, thereby improving chloride transport across cell membranes. This mechanism helps restore proper fluid balance in affected tissues like the lungs and pancreas. Ivacaftor was developed by Vertex Pharmaceuticals and approved by regulatory agencies including the FDA in 2012[1][3][5].

Brand names

Kalydeco

Other names

VX-440VX440VX 440