FDA approves Kalydeco (Vertex Pharma) for Cystic Fibrosis

The FDA has approved Kalydeco(ivacaftor)from Vertex Pharma, the first medicine to treat the underlying cause of Cystic Fibrosis (CF), a rare, genetic disease. Kalydeco is 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 (CFTR) gene. Approximately 1,200 people in the United States , or 4 percent of those with CF, are believed to have this mutation. The company has established a financial assistance and patient support program to help get Kalydeco to eligible patients for whom it is prescribed. Kalydeco was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. , the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.Kalydeco is filed for approval in the EU.