tezacaftor-vx-661

Description: Tezacaftor (formerly VX-661; trade name: Symdeko) is a second F508del CFTR corrector that is able to help CFTR protein reach the cell surface. VX-661 is potentially useful for treatment of cystic fibrosis disease. In Feb 2018, Tezacaftor was approved by FDA to treat cystic fibrosis in patients age 12 years and older. Cystic fibrosis (CF) is a genetic disease caused by defects in the CF transmembrane regulator (CFTR) gene, which encodes an epithelial chloride channel. The most common mutation, delta508CFTR, produces a protein that is misfolded and does not reach the cell membrane. VX-661 can correct trafficking of delta508CFTR and partially restore chloride channel activity.

References: Sci Transl Med. 2014 Jul 23; 6(246):246ra97.