- SYMDEKO is Vertex’s third medicine to treat
the underlying cause of CF -
- SYMDEKO to begin shipping to pharmacies this
week -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) approved
SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the
underlying cause of cystic fibrosis (CF) in people ages 12 and
older who have two copies of the F508del mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene or who
have at least one mutation that is responsive to
tezacaftor/ivacaftor. SYMDEKO is Vertex’s third medicine approved
to treat the underlying cause of CF. Vertex is ready to launch
SYMDEKO and will begin shipping it to pharmacies in the United
States this week.
This press release features multimedia. View
the full release here:
http://www.businesswire.com/news/home/20180212006334/en/
“Today is an exciting day for the CF community. The approval of
SYMDEKO, our third disease-modifying CF medicine, offers many
patients an important new treatment option,” said Jeffrey
Leiden, M.D., Ph.D., Vertex's Chairman, President and Chief
Executive Officer. “This approval is an important milestone in our
journey to treat every person with CF, and we remain committed to
urgently advancing our efforts to develop new medicines that treat
the underlying cause of CF for the many people still waiting.”
In November 2017, the New England Journal of Medicine published
the results of two Phase 3 studies of SYMDEKO. These studies, named
EVOLVE and EXPAND, enrolled approximately 750 people with CF ages
12 and older with two copies of the F508del mutation or
with one F508del mutation and one mutation that results in residual
CFTR function. Across both studies, patients treated with SYMDEKO
experienced statistically significant and clinically meaningful
improvements in lung function and other measures of disease, with a
favorable safety profile. The most common adverse events,
regardless of treatment group, included infective pulmonary
exacerbation and cough. The first data from the ongoing EXTEND
rollover study, also presented in November, show that the lung
function improvements and the safety and tolerability profiles seen
in EVOLVE and EXPAND were sustained for up to 48 total weeks of
SYMDEKO treatment.
“We’ve already seen the significant impact that
disease-modifying medicines can have on patients and are incredibly
pleased that there is now a third treatment option that enables
more patients to benefit from CFTR modulation,” said Patrick Flume,
M.D., Director of the Medical University of South Carolina Cystic
Fibrosis Center and Principal Investigator for the EXTEND study.
“In particular, SYMDEKO is an important treatment option for
patients who either never started or discontinued ORKAMBI, and it
also provides increased benefit over KALYDECO alone for patients
with residual function mutations.”
The European Medicines Agency (EMA) has validated the Marketing
Authorization Application (MAA) for the tezacaftor/ivacaftor
combination. The company expects approval in the EU in the second
half of 2018.
Helping Patients Access SYMDEKO
The people who work at Vertex understand that medicines can
only help patients who can get them. The Vertex Guidance &
Patient Support (Vertex GPS™) program provides a team of Vertex
employees dedicated to helping eligible patients who have been
prescribed our medicines within their labeled
indications understand their insurance benefits and the
resources that are available to help them.
Vertex also offers a co-pay assistance program for patients
with commercial insurance coverage and a free medicine program for
qualifying patients who are uninsured and who meet certain income
and other eligibility criteria. More information is available by
visiting www.VertexGPS.com or by calling
1-877-752-5933.
About CF
Cystic Fibrosis (CF) is a rare, life-shortening 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 test, or genotyping test, lead to CF by
creating non-working or too few CFTR proteins at the cell surface.
The defective function or absence of CFTR protein 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 SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor)
Some mutations result in CFTR protein that is not processed or
folded normally within the cell, and that generally does not reach
the cell surface. SYMDEKO is a combination of tezacaftor and
ivacaftor. Tezacaftor is designed to address the trafficking and
processing defect of the CFTR protein to enable it to reach the
cell surface where ivacaftor can increase the amount of time the
protein stays open.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR SYMDEKO™
(tezacaftor/ivacaftor and ivacaftor) tablets
SYMDEKO is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 12 years and older who have
two copies of the F508del mutation, or who have at least one
mutation in the CF gene that is responsive to treatment with
SYMDEKO. Patients should talk to their doctor to learn if they have
an indicated CF gene mutation. It is not known if SYMDEKO is safe
and effective in children under 12 years of age.
Patients should not take SYMDEKO if they take certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; St. John’s wort.
Before taking SYMDEKO, patients should tell their doctor if
they: have or have had liver problems; have kidney problems;
are pregnant or plan to become pregnant because it is not known if
SYMDEKO will harm an unborn baby; are breastfeeding or planning to
breastfeed because it is not known if SYMDEKO passes into breast
milk.
SYMDEKO may affect the way other medicines work, and other
medicines may affect how SYMDEKO works. Therefore, the dose of
SYMDEKO may need to be adjusted when taken with certain medicines.
Patients should especially tell their doctor if they take
antifungal medicines such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
SYMDEKO may cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
requires alertness until they know how SYMDEKO affects them.
Patients should avoid food or drink that contains
grapefruit or Seville oranges while they are taking SYMDEKO.
SYMDEKO can cause serious side effects, including:
High liver enzymes in the blood, which have been reported
in people treated with SYMDEKO or treated with ivacaftor alone. The
patient’s doctor will do blood tests to check their liver before
they start SYMDEKO, every 3 months during the first year of taking
SYMDEKO, and every year while taking SYMDEKO. Patients should call
their doctor right away if they have any of the following symptoms
of liver problems: pain or discomfort in the upper right stomach
(abdominal) area; yellowing of the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine.
Abnormality of the eye lens (cataract) in some children
and adolescents treated with SYMDEKO or with ivacaftor alone. If
the patient is a child or adolescent, their doctor should perform
eye examinations before and during treatment with SYMDEKO to look
for cataracts.
The most common side effects of SYMDEKO include headache,
nausea, sinus congestion, and dizziness.
These are not all the possible side effects of SYMDEKO.
Please click here to see the full Prescribing
Information for SYMDEKO.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious and life-threatening diseases. In addition to clinical
development programs in CF, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada and Australia. Vertex is consistently
recognized as one of the industry's top places to work, including
being named to Science magazine's Top Employers in the life
sciences ranking for eight years in a row. For additional
information and the latest updates from the company, please visit
www.vrtx.com.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation. KALYDECO®
(ivacaftor), ORKAMBI®(lumacaftor/ivacaftor), SYMDEKO™
(tezacaftor/ivacaftor and ivacaftor), VX-440, VX-152, VX-659 and
VX-445 were discovered by Vertex as part of this collaboration.
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 statements by Dr. Leiden in the second
paragraph and Dr. Flume in the fourth paragraph of this press
release and statements regarding (i) the timing of shipping SYMDEKO
in the United States; and (ii) the anticipated timing of approval
by the EMA. 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 SYMDEKO in the United
States, obtaining approval and commercializing tezacaftor/ivacaftor
in Europe, developing additional medicines to treat cystic fibrosis
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)
View source
version on businesswire.com: http://www.businesswire.com/news/home/20180212006334/en/
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comorNorth America:Megan
Goulart, + 1-617-341-6992orEurope & Australia:Rebecca Hunt, +44
7718 962 690
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Feb 2024 to Mar 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Mar 2023 to Mar 2024