SILVER SPRING, Md. — The Food and Drug Administration has approved a drug made by Vertex Pharmaceuticals for treating a rare form of cystic fibrosis, the agency said Tuesday.
The FDA announced the approval of Kalydeco (ivacaftor) for treating patients ages 6 years and older with CF who have a mutation called G551D in the cystic fibrosis transmembrane regulator gene.
CF is a serious disorder that leads to an early death by causing thick mucus to build up in the lungs, digestive tract and other parts of the body, eventually leading to respiratory and digestive problems and such complications as infections and diabetes. Of the 30,000 people in the United States with the disease, about 1,200 are estimated to have the G551D mutation.
"Kalydeco is an excellent example of the promise of personalized medicine — targeted drugs that treat patients with a specific genetic makeup," FDA commissioner Margaret Hamburg said.