The European Medicines Agency (EMA) has recommended Vertex Pharmaceuticals' Kalydeco (ivacaftor), an orphan-designated medicine, for the treatment of cystic fibrosis in patients aged six years and above who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene.
Cystic fibrosis is a rare, life-threatening genetic disorder caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene, which regulates salt and water transport in the body.
The FDA-approved Kalydeco acts by targeting the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein.
The agency's Committee for Medicinal Products for Human Use (CHMP) reviewed Kalydeco under accelerated assessment to enhance patient access to new medicines that are of public health interest.
The CHMP opinion was based on positive findings from two global Phase 3 studies, in which Kalydeco demonstrated improvements in breathing and other measures of disease, as well as showing sustained improvements in lung function, weight gain and certain quality of life measurements, compared to those on placebo.
In addition, people administered with Kalydeco were 55% less likely to have pulmonary exacerbations, or periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits, than those who received placebo.
Previous clinical studies demonstrated that Kalydeco improved pulmonary function in cystic fibrosis patients with the specific G551D-CFTR mutation.
European Cystic Fibrosis Society president and Kalydeco investigator Stuart Elborn said: "Kalydeco is a fundamentally different approach to the way we treat cystic fibrosis because it targets the underlying cause of the disease. In clinical trials, Kalydeco helped people with a specific genetic mutation breathe more easily, gain weight and generally feel better."
The CHMP's positive opinion will be reviewed by the European Commission to approve Kalydeco in the European Union.