Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor
and ivacaftor), which can treat the underlying cause of cystic
fibrosis (CF) in ~90% of people with the disease, will be presented
at the 2023 North American Cystic Fibrosis Conference (NACFC)
taking place November 2-4, 2023 in Phoenix, Arizona. Presentations
will highlight results from two open-label extension studies
demonstrating the long-term clinical benefits and safety profile of
TRIKAFTA® among CF pediatric populations.
Vertex will present interim data on TRIKAFTA® from its longest
pediatric follow-up study to date. This interim analysis through
Week 144 of a 192-week open-label extension study of TRIKAFTA® in
children 6 years of age and older with CF and at least one F508del
allele showed sustained improvements in CFTR function and
respiratory symptoms following the pivotal study. In addition,
there was no evidence of a decline in lung function during the
treatment period of 3.5 years in the pivotal and extension studies.
The data also showed a continued favorable safety profile, with no
new safety findings (Poster #133).
In addition, 48-week interim results from a 192-week open-label
study of TRIKAFTA® in children 2 years of age and older with cystic
fibrosis and at least one F508del allele showed that the clinical
benefits from the parent pivotal study were sustained, including
improvements in lung function and CFTR function. There was also
evidence suggesting continued improvement in exocrine pancreatic
function with ongoing use of TRIKAFTA®. TRIKAFTA® continued to be
generally safe and well tolerated through an additional 48 weeks of
treatment (Poster #137).
“TRIKAFTA has fundamentally changed the course of CF treatment,
and the data presented at this year’s NACFC is further evidence of
the long-term benefits it brings to children with this disease,”
said Carmen Bozic, M.D., Executive Vice President, Global Medicines
Development and Medical Affairs, and Chief Medical Officer at
Vertex. “We remain committed to developing medicines that can be
given to people with CF as young as possible, because we now know
treating the disease early in life can slow disease
progression.”
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 88,000 people globally. CF is a progressive,
multi-organ disease that affects the lungs, liver, pancreas, GI
tract, sinuses, sweat glands and reproductive tract. CF is caused
by a defective and/or missing CFTR protein resulting from certain
mutations in the CFTR gene. Children must inherit two defective
CFTR genes — one from each parent — to have CF, and these mutations
can be identified by a genetic test. While there are many different
types of CFTR mutations that can cause the disease, the vast
majority of people with CF have at least one F508del mutation. CFTR
mutations lead to CF by causing CFTR protein to be defective or by
leading to a shortage or absence of CFTR protein at the cell
surface. The defective function and/or absence of CFTR protein
results in poor flow of salt and water into and out of the cells in
a number of organs. In the lungs, this leads to the buildup of
abnormally thick, sticky mucus, chronic lung infections and
progressive lung damage that eventually leads to death for many
patients. The median age of death is in the 30s, but with
treatment, projected survival is improving.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor)
INDICATIONS AND USAGE
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a
prescription medicine used for the treatment of cystic fibrosis
(CF) in patients aged 2 years and older who have at least one copy
of the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene or another mutation that is
responsive to treatment with TRIKAFTA. Patients should talk to
their doctor to learn if they have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under
2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, patients should tell their doctor
about all of their medical conditions, including if they: are
allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney
problems, have or have had liver problems, are pregnant or plan to
become pregnant because it is not known if TRIKAFTA will harm an
unborn baby, or are breastfeeding or planning to breastfeed because
it is not known if TRIKAFTA passes into breast milk.
Patients should tell their doctor about all the medicines
they take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. TRIKAFTA may affect
the way other medicines work, and other medicines may affect how
TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when
taken with certain medicines. Patients should ask their doctor or
pharmacist for a list of these medicines if they are not sure.
Patients should especially tell their doctor if they take:
antibiotics such as rifampin or rifabutin; seizure medicines such
as phenobarbital, carbamazepine, or phenytoin; St. John’s wort;
antifungal medicines including ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; antibiotics including
telithromycin, clarithromycin, or erythromycin.
Patients should avoid food or drink that contains
grapefruit while taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in patients
with severe liver disease that can be serious and may require
transplantation. Liver damage has also happened in patients without
liver disease.
High liver enzymes in the blood, which is a common side
effect in patients treated with TRIKAFTA. These can be
serious and may be a sign of liver injury. The patient’s doctor
will do blood tests to check their liver before they start
TRIKAFTA, every 3 months during the first year of taking TRIKAFTA,
and every year while taking TRIKAFTA. 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.
Serious allergic reactions have happened to patients who
are treated with TRIKAFTA. Call your healthcare provider or go to
the emergency room right away if you have any symptoms of an
allergic reaction. Symptoms of an allergic reaction may include:
rash or hives; tightness of the chest or throat or difficulty
breathing; swelling of the face, lips and/or tongue; difficulty
swallowing; and light-headedness or dizziness.
Abnormality of the eye lens (cataract) has been noted in
some children and adolescents treated with TRIKAFTA. If the patient
is a child or adolescent, their doctor should perform eye
examinations before and during treatment with TRIKAFTA to look for
cataracts.
The most common side effects of TRIKAFTA include
headache, upper respiratory tract infection (common cold) including
stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash,
increase in liver enzymes, increase in a certain blood enzyme
called creatine phosphokinase, flu (influenza), inflamed sinuses,
and increase in blood bilirubin.
Patients should tell their doctor if they have any side effect
that bothers them or that does not go away. These are not all the
possible side effects of TRIKAFTA. For more information, patients
should ask their doctor or pharmacist.
Please click here to see the full U.S.
Prescribing Information for TRIKAFTA
(elexacaftor/tezacaftor/ivacaftor and ivacaftor).
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
clinical pipeline of investigational therapies across a range of
modalities in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, acute and neuropathic
pain, type 1 diabetes and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international headquarters in London. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia and Latin America.
Vertex is consistently recognized as one of the industry's top
places to work, including 14 consecutive years on Science
magazine's Top Employers list and one of Fortune’s 100 Best
Companies to Work For. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
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, without limitation, statements by Carmen Bozic,
M.D., in this press release, and our plans to present data on
TRIKAFTA® at the NACFC, including long-term clinical benefits and
safety profile in CF pediatric populations. 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 risks and uncertainties that could cause actual events or
results to differ materially from those expressed or implied by
such forward-looking statements. Those risks and uncertainties
include, among other things, that data from the company's research
and development programs may not support registration or further
development of its compounds due to safety, efficacy, and other
risks listed under the heading “Risk Factors” in Vertex's most
recent annual report and subsequent quarterly reports filed with
the Securities and Exchange Commission at www.sec.gov and available
through the company's website at www.vrtx.com. You should not place
undue reliance on these statements, or the scientific data
presented. 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: https://www.businesswire.com/news/home/20231102681262/en/
Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com or Manisha Pai: +1 617-961-1899 Media:
mediainfo@vrtx.com or U.S.: +1 617-341-6992 or Heather Nichols: +1
617-839-3607
Vertex Pharmaceuticals (NASDAQ:VRTX)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024