The Food and Drug Administration has approved the drug Kalydeco for the treatment of a rare form of cystic fibrosis in patients 6 years and older, the agency announced Tuesday.
The drug, manufactured by Vertex Pharmaceuticals, is targeted specifically at helping patients who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator gene. About 1,200 of the 30,000 people in the United States affected by cystic fibrosis, a disease that causes a thick mucus buildup in the lungs and digestive tract, are believed to have the G551D mutation, according to the FDA.
“Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup,” FDA Commissioner Margaret A. Hamburg said in a statement announcing the FDA’s approval. “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”
The FDA approved the drug after just three months of consideration under its expedited priority program that identifies rare diseases affecting fewer than 200,000 people. The decision was made based on an analysis of two 48-week studies involving 213 patients, with one focused on patients 12 years and older and the other on patients 6 years to 11 years old.
In both cases, Kalydeco, taken two times a day in pill form, was found to significantly improve and sustain lung function.