bamocaftor

Bamocaftor (VX-659) is a next-generation small molecule corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It was developed by Vertex for use in combination with tezacaftor and ivacaftor as a triple therapy for cystic fibrosis (CF), particularly targeting patients with at least one F508del mutation in the CFTR gene. Bamocaftor acts by correcting the misfolded CFTR protein, facilitating its proper trafficking to the cell surface, thereby improving chloride transport across epithelial cells. In clinical trials, this triple combination demonstrated significant improvements in lung function and other clinical outcomes compared to placebo or dual therapy. The drug reached Phase 3 development but was not ultimately submitted for approval; instead, Vertex advanced another triple combination regimen using elexacaftor[1][3][4][6][8].

Other names

bamocaftor2204245-48-5