-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 Europe ages 12 and older have two copies of the F508del
mutation-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the submission of 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) for a fully co-formulated combination of
lumacaftor (400mg q12h) and ivacaftor (250mg q12h) for people with
cystic fibrosis (CF) ages 12 and older who have two copies of the
F508del mutation in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene. There are approximately 22,000 people with
CF ages 12 and older who have two copies of the F508del mutation
in North America, Europe and Australia, including
approximately 8,500 in the United States and 12,000
in Europe.
“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 Jeffrey
Chodakewitz, M.D., Executive Vice President and Chief Medical
Officer at Vertex. “Today’s submissions represent important
progress toward our ongoing efforts to develop new medicines for
the vast majority of people with cystic fibrosis, and we look
forward to working closely with regulatory agencies to bring this
treatment to eligible patients as quickly as possible.”
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 European Committee for Medicinal
Products for Human Use (CHMP) has granted Vertex’s request for
Accelerated Assessment of the MAA, which is given to new medicines
of major public health interest and shortens the review time from
approximately 210 to 150 days for the CHMP to give an opinion
following the start of the review. The CHMP opinion is then
reviewed by the European Commission, which generally issues a final
decision within three months. If approved, Vertex would then begin
the country-by-country reimbursement approval process. Both
applications seek approval for a fully co-formulated combination
treatment dosed as two tablets every 12 hours (four tablets
daily).
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 FDA. Vertex will also work with regulatory
authorities outside the United States toward implementing
additional expanded access programs in other countries, with a goal
of opening programs for eligible patients in the second quarter of
2015.
For more information, please contact Vertex Medical Information
(U.S.: 1-877-634-8789 or medicalinfo@vrtx.com; outside the U.S.:
vertexmedicalinfo@vrtx.com).
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 North America, Europe and Australia, there
are approximately 22,000 people ages 12 and older who have two
copies of the F508del mutation.
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.
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, Dr. Chodakewitz's statements in the
second paragraph of the press release, and the information provided
regarding (i) Vertex’s NDA submission to the FDA and MAA submission
to the EMA, (ii) Vertex’s request for priority review and (iii)
Vertex's planned compassionate use program and Phase 3b study.
While Vertex believes the forward-looking statements contained in
this press release are accurate, these forward-looking statements
represent the company's beliefs only as of the date of this press
release and 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 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)
Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge,
617-341-6108orKelly Lewis, 617-961-7530orMedia:
mediainfo@vrtx.comU.S.: Zach Barber, 617-341-6992orEurope: Megan
Goulart, +41 22 593 6066
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