Kalydeco Cystic Fibrosis
Kalydeco (ivacaftor) is a cystic fibrosis transmembrane
conductance regulator (CFTR) potentiator. Cystic fibrosis is caused by
mutations in a gene that encodes for the CFTR protein that regulates ion
(such as chloride) and water transport in the body. The defect in
chloride and water transport results in the formation of thick mucus
that builds up in the lungs, digestive tract and other parts of the body
leading to severe respiratory and digestive problems.Kalydeco
was specifically approved for the treatment of cystic fibrosis in
patients age 6 years and older who have a G551D mutation in the CFTR
gene. If the patient?s genotype is unknown, an FDA-cleared CF mutation
test should be used to detect the presence of the G551D mutation.
Kalydeco
is supplied as a tablet for oral administration. The recommended dose
of Kalydeco for both adults and pediatric patients age 6 years and older
is one 150 mg tablet taken orally every 12 hours (300 mg total daily
dose).
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