FDA approves CF treatment Full Text
Drug Topics, 05/15/2012
Cystic fibrosis (CF), a disorder of the lungs and other organs that affects about 30,000 people in the United States, is caused by mutations in the gene encoding the CFTR protein. On January 31, FDA approved ivacaftor (Kalydeco) to treat CF in patients 6 years of age and older who have a G551D mutation in the CFTR gene. Approximately 4% of CF patients, roughly 1,200 people, have this mutation.