FDA Approves Vertex Pharmaceutical's Kalydeco For Cystic Fibrosis
By Cyndi Root
Vertex Pharmaceuticals, Inc. announced via press release that the Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor). The drug is indicated for people with cystic fibrosis (CF) ages six and older who have one of eight mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Robert Kauffman, M.D. Ph.D., Senior Vice President and Co-Chief Medical Officer at Vertex, said, "We believe that Kalydeco has the potential to help more people with CF, and today's approval is an important step toward that goal. As we progress over the coming year, we look forward to data from multiple other ongoing studies that are designed to evaluate whether additional people with CF may benefit from Kalydeco."
Cystic Fibrosis
Cystic fibrosis (CF) is a hereditary disease affecting adults and children. The chronic disease affects the digestive system and the lungs. Mucus clogs the lungs and leads to infections that can be life threatening. The mucus can also stop enzymes in the pancreas that break down and metabolize food. About 30,000 people in the U.S. and 70,000 worldwide have the disease. Before life-saving technologies and modern medicine, few people with CF lived, but now many live into their forties and beyond. CF is caused by missing or defective CFTR proteins, which cause deficits in water and salt in and out of the lungs and other organs.
Kalydeco
Kalydeco (ivacaftor) improves the CFTR channel thereby increasing chloride transport. The drug was approved with a Breakthrough Therapy designation in 2012 for people with CF ages six and older with at least one copy of the G551D mutation. With the latest approval, Kalydeco is now indicated for certain mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D. The FDA approved the drug in part due to the results gathered from a Phase 3 study. In the placebo-controlled study of 39 people with CF who had one of the indicated mutations, results showed statistically significant improvements in lung function. Kalydeco is also approved by the European Medicines Agency, Health Canada, and the Therapeutic Goods Administration in Australia.
About Vertex
Vertex Pharmaceuticals is an American company with operations in Canada, Europe, and Australia. The FDA approved the company’s first drug, for hepatitis C, in 2011. It is developing therapies for rheumatoid arthritis and influenza.