-More than 1,200 children are newly eligible
for a medicine that could treat the underlying cause of their
disease-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted approval for the
label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
combination regimen with ivacaftor for the treatment of children
with cystic fibrosis (CF) ages 2 through 5 years old who have at
least one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
“In addition to data from clinical trials, long-term and
real-world data have demonstrated the significant clinical benefit
of KAFTRIO in eligible people living with CF, and today’s news
means that young children across Europe can now benefit from this
important medicine,” said Carmen Bozic, M.D., Executive Vice
President, Global Medicines Development and Medical Affairs, and
Chief Medical Officer, Vertex.
“As CF starts in early childhood and is a progressive disease,
it is important to treat people with CF as early as possible. With
the approval of KAFTRIO for children as young as 2 years, we can
now treat young children with a medicine that has the potential to
slow disease progression by addressing the underlying cause of the
disease,” said Professor Marcus A. Mall, M.D., Head of the
Department of Pediatric Respiratory Medicine, Immunology and
Critical Care Medicine at Charité Universitätsmedizin Berlin.
As a result of existing reimbursement agreements in Austria,
Denmark, Ireland, Norway, Latvia, and Sweden, eligible patients in
these countries will have access to the expanded indication for
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination
regimen with ivacaftor shortly following regulatory approval by the
European Commission. Vertex will continue to work with
reimbursement authorities across the European Union to ensure
access for all eligible patients. In the U.K., following MHRA
approval on November 15, 2023, and as a result of the existing
reimbursement agreement between Vertex and the NHS, children ages 2
years and above in the U.K. have access to this expanded indication
for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination
regimen with ivacaftor.
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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in
Combination With Ivacaftor In people with certain types of
mutations in the CFTR gene, the CFTR protein is not processed or
folded normally within the cell, and this can prevent the CFTR
protein from reaching the cell surface and functioning properly.
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with
ivacaftor is an oral medicine designed to increase the quantity and
function of the CFTR protein at the cell surface. Elexacaftor and
tezacaftor work together to increase the amount of mature protein
at the cell surface by binding to different sites on the CFTR
protein. Ivacaftor, which is known as a CFTR potentiator, is
designed to facilitate the ability of CFTR proteins to transport
salt and water across the cell membrane. The combined actions of
ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus
from the airways.
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with
ivacaftor is approved in the European Union 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 CFTR gene.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
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 approved
medicines that treat the underlying causes of multiple chronic,
life-shortening genetic diseases — cystic fibrosis, sickle cell
disease and transfusion-dependent beta thalassemia — and continues
to advance clinical and research programs in these diseases. Vertex
also 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 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, including,
without limitation, statements made by Carmen Bozic, M.D., and
Professor Marcus A. Mall, M.D., in this press release and
statements regarding the number of children newly eligible for
KAFTRIO, our expectations for patient access to KAFTRIO, including
statements about our efforts to ensure patient access in the EU,
and statements regarding the anticipated benefits of KAFTRIO. 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
risks listed under the heading “Risk Factors” in Vertex's annual
report and in subsequent filings filed with the Securities and
Exchange Commission and available through the company's website at
www.vrtx.com and www.sec.gov. You should not place undue reliance
on these statements. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
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