Vertex outlines progress on cystic fibrosis drug development

USA-based biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX) provided a comprehensive update on recent progress in its R&D activities in cystic fibrosis (CF) in conjunction with the 27th annual North American Cystic Fibrosis Conference (NACFC) in Salt Lake City.

Vertex announced that the TRAFFIC and TRANSPORT Phase III studies of lumacaftor (VX-809) in combination with ivacaftor (its already marketed CF drug Kalydeco) in people with two copies of the F508del mutation are fully enrolled. Data from these studies are expected in mid-2014, and Vertex plans to submit a New Drug Application in the US and a Marketing Authorization Application in Europe in the second half of 2014 for the combination of lumacaftor and ivacaftor.

Vertex also provided updates on multiple ongoing label-expansion studies for ivacaftor, ongoing and planned Phase II combination studies of lumacaftor and ivacaftor and VX-661 and ivacaftor, and research efforts aimed at beginning clinical development of a next-generation corrector. The company also presented additional data from a study of ivacaftor in people with non-G551D gating mutations and data from the PERSIST open-label rollover study will be presented.