CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals Inc. (VRTX) announced Tuesday that FDA has approved Kalydecotm or ivacaftor to treat the underlying cause of cystic fibrosis or CF. Kalydeco is already 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. Cystic fibrosis is a rare, life-threatening genetic disease for which there is no cure. Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have this mutation. Kalydeco got the approval in around three months. The approval is based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain and certain quality of life measurements, compared to those who received placebo.
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