Vertex Pharmaceuticals - Ivacaftor

Ivacaftor

January 31, 2012, Vertex gained FDA approval of the first drug, ivacaftor, to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients over age 6 who have the G551D gene mutation. In the US, 30,000 people have cystic fibrosis. About 4% of those, or 1,200, have the G551D gene mutation. Vertex is marketing the drug at $294,000 a year per patient. Vertex also is studying ivacaftor in combination with another drug (VX-809) for the most common mutation in CF, known as F508del, and expects the first set of results in 2012. Vertex worked for 13 years with the Cystic Fibrosis Foundation to develop the drug.