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Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation
-U.S. submission includes request for Priority Review; Accelerated Assessment has been granted in the EU-
-Approximately 8,500 people in the U.S. and 12,000 in
"The combination of lumacaftor and ivacaftor is the first potential
treatment designed to target the underlying cause of cystic fibrosis in
people with two copies of the F508del mutation, which is the most common
form of the disease," said
In the U.S., the combination of lumacaftor and ivacaftor received
Breakthrough Therapy Designation in late 2012. The U.S. submission
includes a request for Priority Review, which, if granted, would shorten
the FDA's anticipated review time from approximately 12 to 8 months.
The NDA and MAA submissions are based on previously announced data from two global Phase 3 studies, TRAFFIC and TRANSPORT, and the first interim data from the subsequent rollover study in people ages 12 and older who have two copies of the F508del mutation treated with standard-of-care medicines. The TRAFFIC and TRANSPORT studies showed improvements in lung function and other measures of disease, such as pulmonary exacerbations, through 24 weeks of treatment with lumacaftor in combination with ivacaftor. Initial interim data from the rollover study showed that lung function improvements were sustained for 48 total weeks of treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks in rollover study). The combination was generally well tolerated in all three studies. In TRAFFIC and TRANSPORT, the most common adverse events were infective pulmonary exacerbation, cough, headache and increased sputum.
Cystic fibrosis is a rare genetic disease for which there is no cure. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. The defective or missing protein results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Lumacaftor, a CFTR corrector, is designed to address the processing and trafficking defect of the F508del-CFTR protein, increasing the amount of functional protein at the cell surface where ivacaftor, a CFTR potentiator, can further enhance its function.
Expanded Access Programs
In recognition of the immediate needs of some people with CF, Vertex is
working to make the combination of lumacaftor and ivacaftor available to
people ages 12 and older who have two copies of the F508del mutation,
are in critical medical need and meet additional eligibility criteria.
In the U.S., Vertex plans to begin a Phase 3b study for a limited number
of people who have severe lung disease in the first quarter of 2015,
followed by an expanded access program in the second quarter of the
year, pending discussions with the
About the Combination
The combination of lumacaftor and ivacaftor is the first potential
medicine designed to treat the underlying cause of CF in people with two
copies of the F508del mutation, the most common form of the disease. In
Known as a CFTR corrector, lumacaftor aims to address the processing and trafficking defect of the F508del-CFTR protein to enable it to reach the cell surface where the CFTR potentiator, ivacaftor, can further enhance the ion channel function of the CFTR protein. Ivacaftor is designed to help the CFTR channel at the cell surface open more often to improve the transport of salt and water across the cells. In combination, lumacaftor and ivacaftor are believed to help hydrate and clear mucus from the airways.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are more than 1,900 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.
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 Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.
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 Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United
States, Europe, Canada and
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
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