VX-659 + tezacaftor + ivacaftor

VX-659 + tezacaftor + ivacaftor is an investigational triple combination therapy developed for the treatment of cystic fibrosis (CF) in patients with specific mutations in the CFTR gene, particularly those with at least one F508del mutation. The regimen consists of two next-generation CFTR correctors, VX-659 and tezacaftor (also known as VX-661), and a potentiator, ivacaftor (marketed as Kalydeco). Both correctors work by improving the processing and trafficking of defective CFTR protein to the cell surface, while ivacaftor enhances channel opening to increase chloride transport. This mechanism addresses the underlying cause of cystic fibrosis by restoring function to mutated CFTR proteins. Clinical studies have shown significant improvements in lung function and other disease markers compared to placebo or dual combinations[1][2][3][4][5][6].

Other names

triple combination CFTR modulator (VX-659/tezacaftor/ivacaftor)