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

Vertex Announces Phase 2 Results of Combination Treatment in People with One Copy of the F508del CF Mutation

Vertex Pharmaceuticals Inc. announced results from a Phase 2 clinical trial of the cystic fibrosis drug ivacaftor (Kalydeco™) in combination with another potential therapy, lumacaftor, in people with one copy of the F508del mutation, ages 18 and older. In the … Continued

Positive results from Vertex F508del Phase 3 Study with Lumacaftor in Combination with Ivacaftor

Two 24-Week Phase 3 Studies of Lumacaftor in Combination with Ivacaftor Met Primary Endpoint with Statistically Significant Improvements in Lung Function (FEV1) in People with Cystic Fibrosis who have Two Copies of the F508del Mutation Combination of lumacaftor and ivacaftor … 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

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels

BACKGROUND: The most common cystic fibrosis-associated mutation, the deletion of phenylalanine 508 (F508del), results in channels with poor membrane expression and impaired function. VX-770, a clinically approved drug for treatment of CF patients carrying the G551D mutation, and VX-809, a … Continued

Antibacterial properties of the CFTR potentiator ivacaftor

Background Ivacaftor increases CFTR channel activity and improves pulmonary function for individuals bearing a G551D mutation. Because ivacaftor structurally resembles quinolone antibiotics, we tested the hypothesis that ivacaftor possesses antibacterial properties. Methods Bioluminescence, colony forming unit, and minimal inhibitory concentration assays were … 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