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Clinical management of CF » CFTR modulators » VX-809 + ivacaftor

VX-809 + ivacaftor

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:

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.

Click here to visit the clinical trials section of this website to discover more.

  1. Pettit RS. Ann Pharmacother 2012;46:1065–75
  2. Bobadilla JL et al. Hum Mutat 2002;19:575–606
  3. Van Goor F et al. Proc Natl Acad Sci USA 2011;108:18843–8