The Food and Drug Administration (FDA) has approved a drug, called Kalydeco, which is the first to treat the cause of cystic fibrosis and not just the symptoms. The drug is approved for patients age 6 and older with the G551D mutation.
Because Kalydeco helps to fix a single defect in the protein that causes cystic fibrosis, the drug will only help about 1,200 patients who have the rare G551D mutation of the disease. 30,000 Americans have the disease.
Vertex Pharmaceuticals, the maker of Kalydeco, has 2 other drugs in clinical trials that researchers hope will help many more patients with a more common gene defect.
The Cystic Fibrosis Foundation played a major role in the research and development of Kalydeco by investing $75 million. The Foundation will receive royalties from the sales of the drug, costing about $294,000 per year per patient, and reinvest the funds in further research.
Vertex also announced it will give away Kalydeco to anyone who is uninsured and makes under $150,00 per year.
Cystic fibrosis is a genetic disorder that causes thick, sticky mucus to build up in the lungs, digestive tract and other areas of the body. The build up leads to life-threatening lung infections and serious digestion problems. The average life span is about age 37.
