Vertex Presents Long-term Data Showing Orkambi, Kalydeco Potential To Treat CF

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) has presented long-term data demonstrating that Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor) show the potential to modify the progression of cystic fibrosis or CF.

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene.

Vertex presented 12 abstracts at the 30th Annual North American Cystic Fibrosis Conference or NACFC. The presentations include final data from the PROGRESS 96-week extension study of the pivotal 24-week Phase 3 studies of Orkambi in people ages 12 and older with two copies of the F508del mutation (TRAFFIC and TRANSPORT).

Other highlights include an analysis of real-world outcomes in patients treated with Kalydeco using data from the United States Cystic Fibrosis Foundation Patient Registry and United Kingdom Cystic Fibrosis Registry.

Jeffrey Chodakewitz, Executive Vice President and Chief Medical Officer at Vertex said, "The growing body of long-term data for KALYDECO and ORKAMBI indicates that treating the underlying cause of CF with CFTR modulators may modify the progression of this serious and life-shortening disease. These data support our goal to develop increasingly effective combination regimens of CFTR modulators for all people with CF."