-KALYDECO is the first medicine to treat the
underlying cause of CF for people with specific mutations in the
CFTR gene-
-KALYDECO facilitates increased chloride
transport by potentiating the channel-open probability (or gating)
of the CFTR protein-
-The eight additional mutations are present in
approximately 150 people ages six and older in the United
States-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the U.S. Food and Drug Administration (FDA) approved a
supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor)
for people with cystic fibrosis (CF) ages 6 and older who have one
of eight additional mutations in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene. KALYDECO was first approved in
January 2012 for people with CF ages 6 and older who have at least
one copy of the G551D mutation. With the approval of the sNDA,
KALYDECO is now approved for use in people with CF with the
following nine mutations: G551D, G178R, S549N, S549R, G551S,
G1244E, S1251N, S1255P and G1349D. In the United States,
approximately 150 people ages 6 and older have one of the
additional eight mutations for which KALYDECO is now approved.
CF is caused by defective or missing CFTR proteins that result
from mutations in the CFTR gene. 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. Ivacaftor facilitates increased chloride transport by
potentiating the channel-open probability (or gating) of the CFTR
protein.
“We believe that KALYDECO has the potential to help more people
with CF, and today’s approval is an important step toward that
goal," said Robert Kauffman, M.D. Ph.D., Senior Vice President
and Co-Chief Medical Officer at Vertex. “As we progress over the
coming year, we look forward to data from multiple other ongoing
studies that are designed to evaluate whether additional people
with CF may benefit from KALYDECO.”
KALYDECO was granted Breakthrough Therapy designation by the
U.S. FDA in late 2012. The sNDA approval is based on previously
announced data from a Phase 3, two-part, randomized, double-blind,
placebo-controlled, cross-over study of 39 people with CF who had
one of the following mutations: G178R, S549N, S549R, G551S, G1244E,
S1251N, S1255P, G1349D or G970R. The study showed statistically
significant improvements in lung function (FEV1) for people in the
overall study population who received ivacaftor, and the safety
profile was similar to prior Phase 3 studies in people with the
G551D mutation. Based on data from four patients with the G970R
mutation enrolled in the study, the efficacy of KALYDECO in
patients with the G970R mutation could not be established to
support approval in the U.S. Vertex estimates that approximately 10
people with CF have the G970R mutation worldwide, including two
people in the United States.
Data from the study noted above were also used to support
regulatory submissions in Europe, Canada and Australia for approval
of KALYDECO in additional people with CF ages 6 and older. In
Europe and Australia, approximately 250 people with CF have these
additional mutations.
Vertex today reaffirmed its 2014 net revenue guidance for
KALYDECO as provided on January 29, 2014.
About KALYDECOTM (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 the following additional CFTR mutations: G178R,
S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.
KALYDECO was approved by the European Medicines Agency in July
2012, by Health Canada in November 2012 and by the Therapeutic
Goods Administration in Australia in July 2013 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 commercialize
KALYDECO.
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 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. Vertex scientists and our
collaborators are working on new medicines to cure or significantly
advance the treatment of cystic fibrosis, hepatitis C, rheumatoid
arthritis and other life-threatening diseases. In addition to our
clinical development programs, Vertex has more than a dozen ongoing
preclinical 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.
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 only, 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.
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.
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. Kauffman's statements in the
third paragraph of the press release and statements regarding
Vertex’s expectations regarding 2014 KALYDECO net revenues. 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 the company's expectations regarding its
2014 KALYDECO net revenues may be incorrect (including because one
or more of the company's assumptions underlying its revenue
expectations may not be realized), that data from the company's
development programs may not support registration or further
development of its compounds 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:Zach Barber,
617-341-6992mediainfo@vrtx.com
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