The U.S. Food and Drug Administration has approved the first drug to treat the underlying cause of cystic fibrosis, ivacaftor -- marketed under the name Kalydeco. Kalydeco is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator gene.
"Today was a big day for the CF community," said Steven Rowe, M.D., MSPH, associate professor in the Division of Pulmonary, Allergy and Critical Care Medicine at the University of Alabama at Birmingham School of Medicine, who lead multiple studies of the new drug. "I believe this new therapy will substantially improve the lives of CF patients with the G551D mutation, and we hope that with appropriate testing it will also soon also be shown to help patients with other CFTR mutations as well."
The drug targets a defective chloride channel protein in patients with the G551D mutation, enabling that protein to function more efficiently.
Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have the G551D mutation. In the studies, those who were treated with Kalydeco experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain, frequency of hospitalization for CF respiratory problems, and certain quality-of-life measurements, compared to those who received placebo.
The majority of adverse events associated with Kalydeco were mild to moderate. Adverse events commonly observed included headache, upper respiratory tract infection or a common cold, stomach pain and diarrhea.
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