Tezacaftor
Tezacaftor, development code VX-661, is a drug that is sold as a fixed-dose combination with ivacaftor as Symdeko. It is used for the treatment of cystic fibrosis in people six years and older, who have specific gene mutations.
In 2018, the U.S. Food and Drug Administration approved a combination of ivacaftor and tezacaftor; the manufacturer announced a list price of $292,000 per year. The combination of ivacaftor and tezacaftor is being sold as Symdeko.Mechanism of action
Tezacaftor helps move the cystic fibrosis transmembrane conductance regulator protein to the correct position on the cell surface, and is designed to treat people with the F508del mutation.The EVOLVE and EXPAND study findings were published in 2017.History
The U.S. Food and Drug Administration granted the application priority review and granted the approval of Symdeko to Vertex Pharmaceuticals Incorporated.
