New England Journal of Medicine Publishes Data from Two Phase 3 Studies of ORKAMBITM (lumacaftor/ivacaftor) in People with C...
May 17 2015 - 1:32PM
Business Wire
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the New England Journal of Medicine
(NEJM) published data from the two Phase 3 studies of
ORKAMBITM (lumacaftor/ivacaftor), an investigational medicine
designed to treat the underlying cause of cystic fibrosis (CF) in
people ages 12 and older with two copies of the F508del mutation,
the most common form of the disease. The data were published online
today in conjunction with the American Thoracic Society
International Conference (May 15-20, Denver, Colo.) where the data
were presented in a session titled, “Discussion on the Edge: Recent
Pulmonary Research Published in NEJM or JAMA.”
In November 2014, Vertex submitted a New Drug Application
(NDA) to the U.S. Food and Drug Administration (FDA) for
the combination of lumacaftor and ivacaftor in people with CF ages
12 and older who have two copies of the F508del mutation. On May
12th, 2015, the FDA’s Pulmonary Allergy Drugs Advisory Committee
(PADAC) voted 12-1 to recommend that the FDA approve ORKAMBI for
this group of people with CF. The FDA is expected to make a
decision on the ORKAMBI NDA by July 5, 2015.
Cystic fibrosis is a rare genetic disease that is caused by
defective or missing cystic fibrosis transmembrane conductance
regulatory (CFTR) proteins resulting from mutations in
the CFTR gene. The defective or missing proteins result
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.
ORKAMBI is an investigational medicine that is a combination of
lumacaftor, which is designed to increase the amount of functional
protein at the cell surface by addressing the processing and
trafficking defect of the protein, and ivacaftor, which is designed
to enhance the function of the CFTR protein once it reaches the
cell surface.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease
affecting approximately 75,000 people in North
America, Europe and Australia.
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 proteins 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 that eventually leads to death.
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.
About Vertex
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 Australia. For
five years in a row, Science magazine has named Vertex
one of its Top Employers in the life sciences. For additional
information and the latest updates from the company, please
visit www.vrtx.com.
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 the timing of
the potential approval of ORKAMBI as a treatment for patients with
CF twelve years or older who have two copies of the F508del
mutation in their CFTR gene. 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, ORKAMBI for patients with CF twelve
years or older who have two copies of the F508del mutation in their
CFTR gene 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 Securities and Exchange Commission and
available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
617-341-6108orKelly Lewis, 617-961-7530orEric Rojas,
617-961-7205orMedia:US: 617-341-6992mediainfo@vrtx.comorEU:+41 22
593 6066
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