Lumacaftor + Ivacaftor combo success in two Phase III trials for Cystic Fibrosis-Vertex

Vertex Pharmaceuticals Incorporated has announced results from two Phase III studies of lumacaftor in combination with ivacaftor that showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in people ages 12 and older with Cystic Fibrosis (CF) who have two copies (homozygous) of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

All four 24-week combination treatment arms in the studies, known as TRAFFIC and TRANSPORT, met their primary endpoint of mean absolute improvement in ppFEV1 from baseline compared to placebo at the end of treatment. Mean absolute improvements in ppFEV1 of between 2.6 and 4.0 percentage points from baseline compared to placebo were observed across the studies (p?0.0004), with mean relative improvements of 4.3 percent to 6.7 percent (p?0.0007).The combination regimens were generally well tolerated.

Based on these data, Vertex plans to submit regulatory applications for approval in multiple countries, including a New Drug Application (NDA) in the United States and Marketing Authorisation Application (MAA) in Europe , in the fourth quarter of 2014 for people with CF ages 12 and older who have two copies of the F508del mutation.