Vertex Pharmaceuticals Incorporated announced the presentation of long-term data demonstrating that Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor) show the potential to modify the progression of cystic fibrosis (CF).
The presentations given at the 30th Annual North American Cystic Fibrosis Conference (NACFC) 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 (U.S. CFFPR) and United Kingdom Cystic Fibrosis Registry (U.K. CFR).
"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," said Jeffrey Chodakewitz, M.D., executive vice president and chief medical officer at Vertex. "These data support our goal to develop increasingly effective combination regimens of CFTR modulators for all people with CF."
The 12 related abstracts presented at NACFC include data from studies of Vertex medicines and medicines in development.
"These data suggest that the benefits of lumacaftor/ivacaftor are sustained through 96 weeks and indicate that the medicine may modify the progression of CF lung disease by treating its underlying cause," said Michael W. Konstan, M.D., Vice Dean for Translational Research at Case Western Reserve University School of Medicine and Vice Chair for Clinical Research at University Hospitals Rainbow Babies & Children's Hospital who was the principal investigator of the study.
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. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median predicted age of survival for a person born today with CF is 41 years, but the median age of death is 27 years.