-Analysis from TRAFFIC and TRANSPORT extension study of lumacaftor in combination with ivacaftor showed that improvements in lung function and other measures of disease were maintained through 48 weeks in people with cystic fibrosis who have two copies of the F508del mutation-
Lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for the F508del-CFTR mutation (ECFS Abstract WS01.3, oral presentation during Workshop 1 - Strategies to correct CFTR defects.)
One thousand thirty-one people who completed 24 weeks of treatment in either of the Phase 3 studies, TRAFFIC or TRANSPORT, entered the 96-week PROGRESS Phase 3 extension study in which everyone received one of two lumacaftor/ivacaftor combination regimens. An interim analysis was conducted once all patients completed 24 weeks in PROGRESS for a total of 48 total weeks of treatment (48 weeks of treatment with a combination regimen for patients who received a combination regimen in TRAFFIC and TRANSPORT; 24 weeks of treatment with a combination regimen for patients who received placebo in TRAFFIC and TRANSPORT).
These data showed that the initial improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) observed in the 24-week TRAFFIC and TRANSPORT studies among those treated with a lumacaftor/ivacaftor combination were sustained through 48 weeks of treatment across all patients. Reduced rates of pulmonary exacerbations and improvements in body mass index (BMI) and patient-reported respiratory symptoms as measured by the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) were also maintained over 48 weeks. In addition, the pattern and magnitude of response observed after the initiation of combination treatment across all patients who received placebo in TRAFFIC and TRANSPORT and subsequently received a combination regimen in PROGRESS were similar to those seen among patients who received a combination regimen in TRAFFIC and TRANSPORT.
At the time of this analysis, the safety and tolerability results, including the type and frequency of adverse events and serious adverse events, were consistent with those observed in TRAFFIC and TRANSPORT, and no new safety concerns were identified. Over 48 weeks, the most common adverse events were infective pulmonary exacerbation, cough and increased sputum. The incidence of serious adverse events during PROGRESS was generally similar to TRAFFIC and TRANSPORT.
Other data presented at the Conference include:
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 75,000 people in
Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, statements regarding data that will be presented at ECFS.
While Vertex believes the forward-looking statements contained in this
press release are accurate, there are a number of factors that could
cause actual events or results to differ materially from those indicated
by such forward-looking statements. Those risks and uncertainties
include, among other things, that regulatory authorities may not
approve, or approve on a timely basis, lumacaftor in combination with
ivacaftor due to safety, efficacy or other reasons, and other risks
listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the
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