European label extension for Kalydeco in younger patients

The European Commission has granted approval for a label extension on Kalydeco (ivacaftor), the only drug approved in Europe to treat the underlying cause of cystic fibrosis.

The drug’s indications will now include the treatment of children and adolescents with cystic fibrosis (CF), aged six months and older and weighing at least 5kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying CF, says the drug’s developer Vertex Pharmaceuticals (Nasdaq: VRTX), whose shares edged up 1.6% to $275.14 by mid-morning.

“A little over eight years ago, Kalydeco was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” said Dr Reshma Kewalramani, chief executive and president at Vertex, adding: “Since then, it’s been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today’s label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for Kalydeco.”