Ivacaftor works by helping defective proteins function more normally

EU regulators have approved a new small molecule drug for treating cystic fibrosis: ivacaftor, from US biotech Vertex.

Cystic fibrosis is a genetic disease characterised by missing or defective cystic fibrosis transmembrane conductance regulator (CFTR) proteins caused by mutations in the CFTR gene. Ivacaftor is only effective when the disease is caused by the ‘G551D’ mutation, which is the third most common mutation associated with the disease, affecting around 1100 people in Europe. The drug works by helping the defective CFTR proteins function more normally.

Ivacaftor, which is marketed under the brand name Kalydeco, won US approval in January.