-In Europe, approximately 12,000 people with CF
ages 12 and older have two copies of the F508del mutation-
-Positive CHMP Opinion was received in
September 2015-
-Country-by-country reimbursement process will
now begin-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted Marketing
Authorization for ORKAMBI® (lumacaftor/ivacaftor), the first
medicine to treat the underlying cause of cystic fibrosis (CF) in
people ages 12 and older who have two copies of the F508del
mutation. In Europe, there are approximately 12,000 people with CF
ages 12 and older who have two copies of this mutation. Vertex will
now begin the country-by-country reimbursement approval process
across the European Union (EU).
“For people with cystic fibrosis, the disease is a lifelong
battle that becomes progressively more serious with repeated
hospitalization due to lung infections. Until now, people with two
copies of the F508del mutation have only had treatments for the
symptoms and complications of the disease,” said Stuart Elborn,
M.D., Dean, School of Medicine, Dentistry and Biomedical Sciences
at Queen’s University Belfast, and a lead Principal Investigator
for the Phase 3 TRAFFIC study. “The combination of lumacaftor and
ivacaftor represents a step-change in the management of cystic
fibrosis for these patients because it addresses the underlying
cause of the disease. By doing so, it has shown meaningful and
sustained benefits.”
Today’s approval is based on previously announced data from two
24-week global Phase 3 studies, TRAFFIC and TRANSPORT, and
additional interim 24-week data from the subsequent extension
study, PROGRESS, in people ages 12 and older who have two copies of
the F508del mutation and were already being treated with
standard-of-care medicines. In the TRAFFIC and TRANSPORT studies,
which enrolled more than 1,100 patients, those treated with the
combination of lumacaftor and ivacaftor experienced significant
improvements in lung function. Patients also experienced
improvements in body mass index (BMI) and reductions in pulmonary
exacerbations (acute lung infections) including those requiring
hospitalizations and intravenous antibiotic use. Interim data from
PROGRESS showed that these improvements were sustained through 48
total weeks of treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks
in PROGRESS). 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.
The combination of lumacaftor and ivacaftor was generally well
tolerated in all three studies. In TRAFFIC and TRANSPORT, the most
common adverse events included shortness of breath and/or chest
tightness, upper respiratory tract infection (common cold) and
gastrointestinal symptoms (including nausea, diarrhea, or gas). In
the extension study, 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.
“The EU approval of lumacaftor in combination with ivacaftor is
a major milestone in our longstanding efforts to develop new
medicines that treat the underlying cause of the disease for people
with cystic fibrosis,” said Jeffrey Leiden, M.D., Ph.D., Vertex's
Chairman, President and Chief Executive Officer. “This has been 17
years in the making and is the result of a collaborative effort
with the CF community. Our goal now is to help ensure that those
who need this medicine can get it, and to continue our work for the
two out of three people with cystic fibrosis in Europe who still
don’t have a treatment that addresses the underlying cause of their
disease.”
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 cystic fibrosis
transmembrane conductance regulator (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 approximately 2,000 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 function or absence of CFTR proteins in people with CF
results in poor flow of salt and water into and out of the cell in
a number of organs. In the lungs, this leads to the buildup of
abnormally thick, sticky mucus that can cause chronic lung
infections and progressive lung damage in many patients that
eventually leads to death. The median age of death is in the
mid-to-late 20s.
About ORKAMBI® (lumacaftor/ivacaftor) and the
F508del mutation
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.
Patients with two copies of the F508del mutation are easily
identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to
increase the amount of mature protein at the cell surface by
targeting the processing and trafficking defect of the F508del-CFTR
protein, and ivacaftor, which is designed to enhance the function
of the CFTR protein once it reaches the cell surface. It is an oral
pill taken every 12 hours - once in the morning and once in the
evening.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu once
posted.
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. Both
of our approved CF medicines were discovered by Vertex as part of
this collaboration.
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 six
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, as
amended, including the quotes in the second and fifth paragraphs of
this press release and statements regarding the timing of the
availability of ORKAMBI and the country-by-country reimbursement
approval process. While the company 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, risks related to commercializing ORKAMBI in Europe and the
other risks listed under Risk Factors in Vertex's annual report and
quarterly reports filed with the Securities and Exchange Commission
and available through Vertex'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,
+1 617-341-6108orKelly Lewis, +1 617-961-7530orEric Rojas, +1
617-961-7205orMedia:mediainfo@vrtx.comorEurope & Australia: +44
20 3204 5275orNorth America: +1 617-341-6992
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