CHMP recommends EU approval of Kalydeco (Vertex) for Cystic Fibrosis

The EU's CHMP recommends the authorisation of Kalydeco (ivacaftor), from Vertex. Kalydeco is a first-in-class medicine for patients aged six and older who have the specific G551D mutation in the Cystic Fibrosis transmembrane regulator gene. The drug was reviewed under accelerated assessment (in 150 days) and is the first treatment that targets the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein. The drug was approved by the FDA in January.