-In Europe, approximately 250 people ages 6 and
older have one of 8 additional gating mutations-
-KALYDECO is the first medicine to treat the
underlying cause of CF in people with specific non-G551D gating
mutations-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has approved KALYDECO™
(ivacaftor) for people with cystic fibrosis (CF) ages 6 and older
who have one of eight non-G551D gating mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene. Today’s
approval follows the positive opinion from the Committee for
Medicinal Products for Human Use (CHMP) in June 2014. KALYDECO was
first approved in Europe in July 2012 for people with CF ages 6 and
older who have the G551D mutation, which is the most common gating
mutation. The eight additional gating mutations included in today’s
approval are: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P
and G1349D. In Europe, approximately 250 people ages 6 and older
have one of these non-G551D gating mutations.
“Today’s approval in people with additional gating mutations
marks another step toward our goal of helping more people with this
disease,” said Simon Bedson, Senior Vice President and
International General Manager at Vertex. “We are committed to
working closely with the appropriate national authorities to make
KALYDECO available for these patients as soon as possible.”
Today’s approval is based on previously announced data from the
first part of a Phase 3, two-part, randomised, double-blind,
placebo-controlled, cross-over study of 39 people with CF ages 6
and older who have a non-G551D gating mutation. The first part of
the study showed statistically significant improvements in lung
function (FEV1), sweat chloride, body mass index and CFQ-R scores.
Data from the second part of the study were presented at the
European Cystic Fibrosis Society Conference in June 2014 and showed
that these improvements were maintained through 24 weeks of
treatment. The safety profile was similar to prior Phase 3 studies
of KALYDECO in people with the G551D mutation.
“By treating the underlying cause of cystic fibrosis, KALYDECO
has changed the way we treat patients with the most common gating
mutation, G551D. In general, people with other gating mutations
have similarly severe disease to people with the G551D mutation and
have an urgent need for new medicines that address the underlying
cause of the disease,” said Kris De Boeck, M.D., Ph.D., Professor
of Pediatric Pulmonology, University Hospital Gasthuisberg, Leuven,
Belgium, and President-Elect of the European Cystic Fibrosis
Society. “In the Phase 3 study in people with non-G551D gating
mutations, KALYDECO led to rapid, significant and sustained
improvements in lung function and other measures of disease.”
In addition, the CHMP approved the inclusion of data from the
long-term follow-up PERSIST study in the KALYDECO label. PERSIST is
a Phase 3, open-label, 96-week, rollover extension trial that
evaluated the long-term safety and durability of treatment with
KALYDECO by enrolling people ages 6 and older with at least one
copy of the G551D mutation who completed 48 weeks of treatment in
the Phase 3 ENVISION and STRIVE studies (placebo and KALYDECO
treatment groups) and met other eligibility criteria. Results from
PERSIST demonstrated that the safety and efficacy of KALYDECO seen
in the Phase 3 STRIVE and ENVISION trials was maintained through
nearly three years (144 weeks) in G551D patients.
Cystic fibrosis is caused by a defective or missing CFTR protein
that results from mutations in the CFTR gene. CFTR proteins act as
channels at the cell surface that control the flow of salt and
water into and out of the cell. In people with gating mutations,
the CFTR protein at the cell surface is defective and does not work
properly, causing abnormally thick, sticky mucus to build up in the
lungs. The digestive tract and a number of other organs are also
affected.
KALYDECO, an oral medicine known as a CFTR potentiator, helps
the CFTR protein function more normally once it reaches the cell
surface. It targets the abnormal CFTR protein channels and opens
them to allow chloride ions to move into and out of the cell, which
helps thin the mucus so it can hydrate and protect the airways.
About KALYDECO™ (ivacaftor)
KALYDECO (ivacaftor) is the first medicine to treat the
underlying cause of CF in people with specific mutations in the
CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral
medicine that aims to help the CFTR protein function more normally
once it reaches the cell surface, to help hydrate and clear mucus
from the airways. KALYDECO (150mg, q12h) was first 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 and in February 2014 for use in people with CF ages 6 and
older who have one of the following additional CFTR mutations:
G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. In
the European Union, KALYDECO was approved in July 2012 for use in
people with CF ages 6 and older who have at least one copy of the
G551D mutation and in July 2014 for use in people with CF ages 6
and older who have one of eight additional gating mutations,
including G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or
G1349D. In Canada, KALYDECO was first approved in November 2012 for
use in people with CF ages 6 and older who have at least one copy
of the G551D mutation and in June 2014 for use in people with CF
ages 6 and older who have one of the following additional CFTR
mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P,
G1349D or G970R.
KALYDECO is also approved in Australia, New Zealand and
Switzerland for use in people with CF ages 6 and older who have at
least one copy of the G551D mutation in the CFTR gene.
Vertex retains worldwide rights to develop and commercialise
KALYDECO.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO™
(ivacaftor)
Ivacaftor (150 mg tablets) is indicated for the treatment of
cystic fibrosis (CF) in patients age 6 years and older who have a
G551D mutation in the CFTR gene.
In the United States and the European Union, ivacaftor is also
indicated for the treatment of CF in patients age 6 and older who
have one of the following mutations in the CFTR gene: G1244E,
G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. In Canada,
ivacaftor is indicated for these same mutations and additionally
for G970R.
Ivacaftor is not effective in patients with CF with 2 copies of
the F508del mutation (F508del/F508del) in the CFTR gene.
The safety and efficacy of ivacaftor in children with CF younger
than 6 years of age have not been established.
Elevated liver enzymes (transaminases; ALT and AST) have been
reported in patients receiving ivacaftor. It is recommended that
ALT and AST be assessed prior to initiating ivacaftor, every 3
months during the first year of treatment, and annually thereafter.
Patients who develop increased transaminase levels should be
closely monitored until the abnormalities resolve. Dosing should be
interrupted in patients with ALT or AST of greater than 5 times the
upper limit of normal. Following resolution of transaminase
elevations, consider the benefits and risks of resuming ivacaftor
dosing.
Use of ivacaftor with medicines that are strong CYP3A inducers,
such as the antibiotics rifampin and rifabutin; seizure medications
(phenobarbital, carbamazepine, or phenytoin); and the herbal
supplement St. John's Wort, substantially decreases exposure of
ivacaftor and may diminish effectiveness. Therefore,
co-administration is not recommended.
The dose of ivacaftor must be adjusted when used concomitantly
with strong and moderate CYP3A inhibitors or when used in patients
with moderate or severe hepatic disease.
Ivacaftor can cause serious adverse reactions including
abdominal pain and high liver enzymes in the blood. The most common
side effects associated with ivacaftor include headache; upper
respiratory tract infection (the common cold), including sore
throat, nasal or sinus congestion, and runny nose; stomach
(abdominal) pain; diarrhea; rash; and dizziness. These are not all
the possible side effects of ivacaftor. A list of the adverse
reactions can be found in the product labeling for each country
where ivacaftor is approved. Patients should tell their healthcare
providers about any side effect that bothers them or does not go
away.
Please see KALYDECO U.S. Prescribing Information, EU Summary of
Product Characteristics, Canadian Product Monograph, Australian
Consumer Medicine Information and Product Information, Swiss
Prescribing Information and Patient Information, and the New
Zealand Datasheet and Consumer Medicine Information.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening 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 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, 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 four 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, the statements in the second and
fourth paragraphs of this press release. 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, the 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.comorEurope &
Australia:Megan Goulart, +41 22 593 6066orNorth America:Zach
Barber, +1-617-341-6992
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