U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals’ Kalydeco (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Based on this approval, Vertex today increased its guidance for 2017 product revenues of Kalydeco to a range of $740 million to $770 million, the company said in its press release.
“Five years ago, Kalydeco became the first medicine to treat the underlying cause of CF. Since then, we have continued to invest in studies to improve the understanding of how this important medicine may benefit others with this serious and life-shortening disease,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are encouraged by the FDA’s willingness to explore innovative ways to make highly effective medicines like Kalydeco with a well-established safety profile available to more people who are in urgent need. We will continue to work closely with the FDA to bring Kalydeco to more people with responsive mutations who are still in need as rapidly as possible.”