Your money or your life? - Kalydeco®: a milestone towards the “cure” of cystic fibrosis From February 1st 2016 on, Ivacaftor (KALYDECO™) will be reimbursed in Belgium for the treatment of cystic fibrosis (CF) in patients aged 6 years and older carrying at least one of nine well characterized gating (Class III) cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations, which account for approximately 3% of Belgian CF patients. In most of these patients, this very expensive oral drug (248,000 €/year) substantially improves lung function, lowers sweat chloride levels, and improves body weight by targeting the primary defect, thus representing a fundamental shift in the way the disease is managed. However, clinicians should be aware of the drug's potential for numerous drug interactions.
Key words
Cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR), ivacaftor
