Vertex Receives European Approval for KALYDECO (ivacaftor) in Eight Non-G551D Gating Mutations

 Vertex Pharmaceuticals Incorporated today announced that the European Commission has approved KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Today’s approval follows … Continued

Proof-of-Concept Study of Ivacaftor Monotherapy Showed Improvements in Lung Function After Two Weeks of Treatment in People with Cystic Fibrosis who have a Residual Function Mutation

Data are consistent with in vitro observations in residual function mutations showing that ivacaftor improved CFTR activity 8-week open-label period showed improvements in lung function Data support plans to initiate a Phase 3 study of ivacaftor in people with CF … Continued

US Food and Drug Administration Approves Kalydeco for Use in Eight Additional Mutations that Cause Cystic Fibrosis

-KALYDECO is the first medicine to treat the underlying cause of CF for people with specific mutations in the CFTR gene- -KALYDECO facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein- -The eight additional … Continued

Vertex Announces Recent Progress and Upcoming Milestones in Research and Development Programs for Cystic Fibrosis

Enrollment complete for TRAFFIC and TRANSPORT Phase 3 studies of lumacaftor (VX-809) in combination with ivacaftor for people with two copies of the F508del mutation (homozygous); data expected in mid-2014 12-week Phase 2 study of VX-661 in combination with ivacaftor … Continued