WASHINGTON, D.C. - Congressman Edward J. Markey (D-Mass.), co-Chair of the Congressional Cystic Fibrosis Caucus, released the following statement after the Federal Drug Administration’s announcement today that it has approved a new drug to treat cystic fibrosis (CF) – Kalydeco – manufactured by Cambridge-based Vertex Pharmaceuticals, Inc. In Massachusetts, 840 children and adults are afflicted with cystic fibrosis.
Vertex’s new treatment is an important step toward ensuring that one day ‘CF’ will stand for ‘Cured Forever’, and children will have to turn to the history books to learn that there ever was a disease called cystic fibrosis,” said Rep. Markey. “I congratulate Vertex Pharmaceuticals for gaining this approval. It is just one example of Massachusetts’s leadership in the health and biotech industries that is driving innovation, job creation and economic growth.”
Cystic fibrosis affects approximately 30,000 in the United States, with an average incidence of about 1,000 new cases annually. The defective gene that causes the disease – known as CFTR – has more than 1,800 mutations. Kalydeco is a personalized medicine targeting the G551D gating mutation (approximately 4 percent of CF patients, or about 1,200 people in the U.S.). It is also the first drug approved that targets the underlying cause of CF, not simply the symptoms.
CF is a life-limiting genetic disorder that reduces the flow of salt and water across cell membranes leading to the buildup of abnormally thick, sticky mucus in the lungs that can cause chronic infections and progressive lung damage. The median predicted age of survival for a person with CF born today is approximately 38 years, but today’s median age of death remains approximately 26 years.
The new treatment was discovered as part of collaboration with the Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.