VX-809 + ivacaftor<< Back to CFTR modulators
VX-809 is a novel CFTR corrector that modulates the folding and trafficking of CFTR protein.1 It is a candidate agent for the treatment for Class II mutations, and in particular, the F508del CFTR mutation that occurs in 90% of patients with CF.2 In vitro experiments have investigated the feasibility of VX-809 as a monotherapy (or in combination with ivacaftor) for the treatment of people with F508del CFTR mutations.
In vitro3 results clearly demonstrated that:
- VX-809 improves the processing of F508del-CFTR and increases its delivery to the cell surface
- The CFTR potentiator, ivacaftor, increases the activity of VX-809 on the cell surface
Several ongoing clinical trials are studying the effectiveness VX-809 as a monotherapy or in combination with ivacaftor for the treatment individuals with the F508del-CFTR mutation.