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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