- Global studies showed significant and sustained improvements in
lung function and other measures of disease among people with a specific
genetic mutation -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that
the European Committee for Medicinal Products for Human Use (CHMP) has
issued a positive opinion by consensus recommending the approval of
KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages 6
and older who have at least one copy of the G551D mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene.
KALYDECO is the first medicine to treat the underlying cause of CF, a
rare, genetic disease caused by defective or missing CFTR proteins
resulting from mutations in the CFTR gene. In people with the
G551D mutation, KALYDECO helps the defective CFTR protein function more
normally. An estimated 1,100 people in Europe have this mutation.
The CHMP opinion was based on positive findings from two global Phase 3
studies in which KALYDECO demonstrated unprecedented improvements in
breathing and other measures of disease for people ages 6 and older with
this specific genetic mutation. People treated with KALYDECO experienced
significant and sustained improvements in lung function, weight gain and
certain quality of life measurements compared to those on placebo. In
addition, people who took KALYDECO were 55 percent less likely to have
pulmonary exacerbations, or periods of worsening in the signs and
symptoms of the disease that often require treatment with antibiotics
and hospital visits, than those who received placebo. Fewer people in
the KALYDECO treatment groups discontinued treatment due to adverse
events than in the placebo groups. The majority of adverse events
associated with KALYDECO were mild to moderate. Adverse events most
commonly observed in those taking KALYDECO included headache, upper
respiratory tract infection (common cold), stomach pain and diarrhea.
"While there has been great progress in cystic fibrosis treatment during
the last few decades, we are still only treating the symptoms and
complications of the disease," said Stuart Elborn, M.D., KALYDECO
investigator and President of the European Cystic Fibrosis Society.
"KALYDECO is a fundamentally different approach to the way we treat
cystic fibrosis because it targets the underlying cause of the disease.
In clinical trials, KALYDECO helped people with a specific genetic
mutation breathe more easily, gain weight and generally feel better."
The CHMP's positive opinion will now be reviewed by the European
Commission, which has the authority to approve medicines for the
European Union. The European Commission generally follows the
recommendation of the CHMP and typically issues marketing approval
within three to four months.
"Since 1998, Vertex has been committed to developing new medicines to
treat the underlying cause of cystic fibrosis," said Peter Mueller,
Ph.D., Chief Scientific Officer and Executive Vice President of Global
Research and Development at Vertex. "KALYDECO represents an important
achievement in this ongoing effort. We look forward to working with the
European Medicines Agency to bring KALYDECO, our first new medicine in
Europe, to people with CF as quickly as possible."
KALYDECO was discovered as part of a collaboration with Cystic Fibrosis
Foundation Therapeutics, Inc., the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 35,000 people in Europe and 70,000 people worldwide.
Today, the median predicted age of survival for a person with CF is
approximately 38 years but the median age of death remains in the
mid-20s. There are more than 1,800 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 absence of working CFTR proteins
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.
In some people, CFTR proteins are present at the cell surface but do not
work properly. One type of this dysfunction is known as the G551D
KALYDECO™ (ivacaftor) is the first treatment to target the underlying
cause of CF. KALYDECO (150mg, q12h) was approved by the U.S. Food and
Drug Administration in January 2012 for use in people with CF ages 6 and
older who have at least one copy of the G551D mutation in the CFTR
gene. Approximately 600 people with CF have started treatment with
KALYDECO since then.
Vertex retains worldwide rights to develop and commercialize KALYDECO.
Indication and Important Safety Information
KALYDECO (150mg, q12h) is a prescription medicine used for the treatment
of cystic fibrosis (CF) in patients age 6 years and older who have a
certain mutation in their CFTR gene called the G551D mutation.
KALYDECO is not for use in people with CF due to other mutations in the CFTR
gene. It is not effective in CF patients with two copies of the F508del
mutation (F508del/F508del) in the CFTR gene.
It is not known if KALYDECO is safe and effective in children under 6
years of age.
KALYDECO should not be used with certain medicines, including the
antibiotics rifampin and rifabutin; seizure medications (phenobarbital,
carbamazepine, or phenytoin); and the herbal supplement St. John's Wort.
KALYDECO can cause serious side effects. High liver enzymes in the blood
have occurred in patients taking KALYDECO as well as those receiving
placebo. Regular assessment is recommended.
The most common side effects associated with KALYDECO include headache;
upper respiratory tract infection (common cold) including sore throat,
nasal or sinus congestion, and runny nose; stomach (abdominal) pain;
diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO. Patients should
tell their healthcare providers about any side effect that bothers them
or doesn't go away.
Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com.
Collaborative History with Cystic Fibrosis Foundation Therapeutics,
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 the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a
cure for cystic fibrosis. The Foundation funds more CF research than any
other organization and nearly every CF drug available today was made
possible because of Foundation support. Based in Bethesda, Md., the
Foundation also supports and accredits a national care center network
that has been recognized by the National Institutes of Health as a model
of care for a chronic disease. The CF Foundation is a donor-supported
nonprofit organization. For more information, visit www.cff.org.
Vertex creates new possibilities in medicine. Our team discovers,
develops and commercializes innovative therapies so people with serious
diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to
cure or significantly advance the treatment of hepatitis C, cystic
fibrosis, rheumatoid arthritis, epilepsy and other life-threatening
Founded more than 20 years ago in Cambridge, MA, we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada.
Today, Vertex has more than 2,000 employees around the world, and Science
magazine named Vertex number one on its 2011 list of Top Employers in
the life sciences.
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 statements
regarding (i) the European Commission generally following the
recommendations of the CHMP and typically issuing marketing approval
within three to four months and (ii) Vertex working with the European
Medicines Agency to bring KALYDECO to people with CF as quickly as
possible. 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 Vertex could experience
unforeseen delays in obtaining approval to market KALYDECO from the
European Commission 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 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.
Vertex Pharmaceuticals Incorporated
Source: Vertex Pharmaceuticals Incorporated
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