KALYDECO® ( ivacaftor ) Approved By FDA For More Than 600 People Ages 2 And Older With Cystic Fibrosis Who Have Certain Residual Function Mutation

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Vertex Pharmaceuticals Incorporated announced that the U.S. FDA has approved KALYDECO® (ivacaftor) for use in more than 600 people with CF ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

This approval was based on Phase 3 clinical data for KALYDECO in these mutations and follows the FDA's approval of KALYDECO in May 2017 for 23 other residual function mutations, which was based on analyses of in vitro data. Both approvals are supported by more than five years of real-world clinical experience that demonstrate KALYDECO's established safety and efficacy profile. Based on today's approval, Vertex increased its guidance for 2017 KALYDECO product revenues to a range of $770 million to $800 million. Vertex's guidance range for total CF product revenues in 2017 is now $1.87 billion to $2.1 billion, including ORKAMBI guidance of $1.1 billion - $1.3 billion. 

"In the five years since KALYDECO became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease." 

CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt (sodium and chloride) and water into or out of cells in a number of organs, including the lungs. The five mutations covered under today's approval (2789+5G— > A, 3272-26A— > G, 3849+10kbC— > T, 711+3A— > G, and E831X) cause CF and result in a moderate loss of chloride transport. People who have these mutations generally experience progressive lung function decline and other complications of the disease. All five of these mutations were evaluated as part of the previously disclosed Phase 3 EXPAND study in which the KALYDECO monotherapy arm met its primary efficacy endpoint and was generally well tolerated. 

KALYDECO is now approved in the U.S. to treat people with CF ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.

 

About Cystic Fibrosis 

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 currently the median age of death is 29 years.

About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for seven years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com