Vertex Pharmaceuticals Incorporated VRTX today announced the
initiation of a global pivotal Phase 3 development program for fixed-dose
combinations of VX-809 (lumacaftor) and ivacaftor in people with cystic
fibrosis (CF) who have two copies (homozygous) of the F508del mutation in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vertex plans
to conduct two 24-week Phase 3 studies to support approval of the combination
of VX-809 and ivacaftor in people with CF ages 12 and older. The studies,
TRAFFIC and TRANSPORT, will each include two treatment groups that will
evaluate VX-809 (600mg QD or 400mg q12h) in combination with ivacaftor (250mg
q12h) compared to a placebo group. Vertex expects to obtain 24-week safety and
efficacy data from both studies and to submit a New Drug Application (NDA) to
the U.S. Food and Drug Administration (FDA) and a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA), pending study
results.
Vertex also plans to conduct a pharmacokinetics and safety study to evaluate
VX-809 in combination with ivacaftor in children with CF ages 6 to 11 who have
two copies of the F508del mutation. The company expects to use the data from
this study for subsequent registration in children ages 6 to 11 in the United
States and is continuing discussions with European regulatory agencies for
patients in this age group.
Vertex will host a conference call for investors and media today, February 26,
2013 at 5:15 p.m. EST, to discuss the company's Phase 3 development plan.
“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,” said Robert Kauffman, M.D.,
Ph.D., Senior Vice President and Chief Medical Officer at Vertex.
“Importantly, these studies will evaluate two doses of VX-809 in combination
with ivacaftor for 24 weeks, and pending data, enable submissions to U.S. and
European regulatory authorities. People with CF are in urgent need of new
treatments, and we are committed to advancing this combination through Phase 3
development as quickly as possible.”
Cystic fibrosis is a rare, life-shortening genetic disease for which there is
no cure. Approximately 70,000 people worldwide have CF, including 30,000 in
the United States and 35,000 in Europe. Globally, nearly half of those with CF
have two copies of the F508del mutation.
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