EMA recommends targeted CF treatment

The European Medicines Agency has recommended approval of the first drug to treat the underlying genetic cause of cystic fibrosis (CF).

Kaydeco (ivacaftor) from Vertex Pharmaceuticals has been recommended by the agency for people with CF aged over 6 who carry the G551D gene mutation.

The drug has the potential to help an estimated 1,100 people in Europe in whom this mutation causes CF.

In people with the G551D mutation, Kalydeco helps the defective protein synthesised by the mutant gene to function more normally.

In two major placebo-controlled phase III studies, patients taking Kalydeco showed sustained improvements in lung function, gained weight and were 55% less likely to suffer exacerbations.

Stuart Elborn, President of the European Cystic Fibrosis Society, said: “While there has been great progress in cystic fibrosis treatment during the last few decades, we are still only treating the symptoms and complications of the disease. Kalydeco is a fundamentally different approach to the way we treat cystic fibrosis because it targets the underlying cause of the disease.”

“Since 1998, Vertex has been committed to developing new medicines to treat the underlying cause of cystic fibrosis,” commented Peter Mueller, Chief Scientific Officer and Executive VP of Global R&D at Vertex. “We look forward to working with the EMA to bring Kalydeco, our first new medicine in Europe, to people with CF as quickly as possible.”

CF is a life-threatening genetic disease affecting 35,000 people in Europe.