Vertex Pharmaceuticals Inc has filed for a New Drug Application (NDA) with the US FDA, and a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for their pipeline combination lumacaftor-ivacaftor drug, indicated for the treatment of cystic fibrosis in patients carrying the F508del CFTR gene alteration.

“The combination of lumacaftor and ivacaftor is the first potential treatment designed to target the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, which is the most common form of the disease,” said Jeffrey Chodakewitz, MD, Executive Vice President and Chief Medical Officer, Vertex.