olacaftor

Olacaftor (VX-440) is a next-generation small molecule CFTR corrector developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis. It acts by improving the processing and trafficking of the F508del-CFTR protein, thereby increasing the amount of functional CFTR protein at the cell surface. When used in combination with other modulators such as tezacaftor (another CFTR corrector) and ivacaftor (a CFTR potentiator), olacaftor aims to further enhance chloride ion transport across epithelial cells, which is defective in cystic fibrosis patients. Clinical studies have evaluated its safety and efficacy both as monotherapy and in dual or triple combinations for people with cystic fibrosis who are homozygous or heterozygous for the F508del mutation[1][5][6][8].

Other names

olacaftorRZ7027HK8FRZ-7027HK8FRZ 7027HK8F