Vertex seeks regulatory approval for lumacaftor/ivacaftor combination for CF

US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX) has filed for marketing approval for a fully co-formulated combination of lumacaftor and ivacaftor with the US Food and Drug Administration and the European Medicines Agency.

The applications are for the use of the combination for people with cystic fibrosis (CF) aged 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are around 22,000 people with CF aged 12 and older who have two copies of the F508del mutation in North America, Europe and Australia, including approximately 8,500 in the USA and 12,000 in Europe.

First potential treatment to target underlying cause of CF