Vertex Submits NDA In U.S. And Europe For Lumacaftor Combined With Ivacaftor

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced the submission of a New Drug Application or NDA to the U.S. Food and Drug Administration or FDA and a Marketing Authorization Application or MAA to the European Medicines Agency or EMA for a fully co-formulated combination of lumacaftor (400mg q12h) and ivacaftor (250mg q12h) for people with cystic fibrosis or CF ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene.

There are around 22 thousand people with CF ages 12 and older who have two copies of the F508del mutation in North America, Europe and Australia, including nearly 8,500 in the U.S. and 12,000 in Europe.

In the U.S., the combination of lumacaftor and ivacaftor received Breakthrough Therapy Designation in late 2012. The U.S. submission includes a request for Priority Review, which, if granted, would shorten the FDA's anticipated review time from nearly 12 to 8 months.