VX-433

VX-433 is an investigational small molecule CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) corrector developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis. As a next-generation corrector, it is designed to address the underlying protein folding and trafficking defects in patients with cystic fibrosis, particularly those carrying the F508del mutation. By facilitating the proper processing and delivery of the CFTR protein to the cell membrane, VX-433 aims to increase the number of functional chloride channels at the cell surface, thereby improving epithelial ion transport. It is currently being evaluated in Phase 1 clinical trials, including studies assessing its safety, pharmacokinetics, and potential drug-drug interactions with metabolic probes like midazolam and bupropion.