On February 26, 2013, Vertex Pharmaceuticals, Inc. announced the initiation of a Phase 3 development program for people with cystic fibrosis who have two copies of the F508del mutation. The program will study various fixed-dose combinations of lumacaftor (VX-809) and ivacaftor over two 24-week Phase 3 studies in CF patients 12 and older. Vertex hopes to obtain 24-week safety and efficacy data from both studies and to submit a New Drug Application to the US Food and Drug Administration and a Marketing Authorization Application to the European Medicines Agency. Researchers will also conduct a pharmacokinetics and safety study to evaluate the same combination in children with CF between the ages of 6 and 11 with two copies of the F508del mutation.
Robert Kauffman, MD, PhD, Senior Vice President and Chief Medical Officer at Vertex said, “This Phase 3 development program is a significant advance in our efforts to develop new medicines that treat the underlying cause of cystic fibrosis for people with the most common type of the disease. Importantly, these studies will evaluate two doses of VX-809 in combination with ivacaftor for 24 weeks, and pending data, enable submissions to US and European regulatory authorities. People with CF are in urgent need of new treatments, and we committed to advancing this combination through Phase 3 development as quickly as possible.”
Source: Vertex Pharmaceuticals Release