Kalydeco is used to treat cystic fibrosis in patients aged six years and above who have the G551D mutation in their gene for the protein called cystic fibrosis transmembrane conductance regulator (CFTR).

Cystic fibrosis is an inherited disease that affects the cells that secrete mucus in the lungs, and the cells that secrete digestive juices from the glands in the gut and pancreas. In cystic fibrosis these secretions become thick, blocking the airways and the flow of digestive juices. This leads to problems with the digestion and absorption of food, resulting in poor growth, and long-term infection and inflammation of the lungs because of excess mucus not being cleared away.

Because the number of patients with cystic fibrosis is low, the disease is considered ‘rare’, and Kalydeco was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 July 2008.

The medicine can only be obtained with a prescription.

Kalydeco is a medicine that contains the active substance ivacaftor. It is available as tablets (150 mg).

Source: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002494/human_med_001575.jsp&mid=WC0b01ac058001d124