VX-828 + deutivacaftor

VX-828 + deutivacaftor is an oral, investigational next-generation triple combination regimen for cystic fibrosis comprising the small molecule CFTR corrector VX-828 and the CFTR potentiator deutivacaftor (VX-118). VX-828 is a next-generation CFTR corrector that helps rescue misfolded and dysfunctional CFTR protein, increasing the amount of protein at the epithelial cell surface. Deutivacaftor is a next-generation CFTR potentiator that enhances the gating and activity of CFTR channels at the cell surface. The regimen targets multiple aspects of CFTR protein folding, trafficking, and function, aiming to maximize restoration of chloride channel activity. This combination is developed by Vertex Pharmaceuticals for cystic fibrosis and is currently in Phase 1 clinical trials, intended to offer improved efficacy over existing modulator therapies by using two correctors (VX-828 plus tezacaftor) and a potentiator (deutivacaftor)[1][2][3][4].

Other names

VX-828 + VX-118VX-828 + deutivacaftor