Therapy Areas: Hereditary Disorders
FDA greenlights Vertex's cystic fibrosis drug KALYDECO
31 January 2012 - US Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) said on Tuesday that KALYDECO (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), has been approved in the US.

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. About 1,200 people in the US, or 4% of those with CF, are believed to have this mutation.

The firm said it has set up a financial assistance and patient support programme to help get KALYDECO to eligible patients for whom it is prescribed.

KALYDECO was discovered as part of a partnership with Cystic Fibrosis Foundation Therapeutics Inc, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
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