- Results from a randomized, placebo-controlled
study of TRIKAFTA® in people with cystic fibrosis with rare,
non-F508del CFTR mutations showed statistically significant and
clinically meaningful improvements in the primary and all secondary
endpoints -
- Interim results of largest real-world study
of TRIKAFTA® showed sustained improvement in lung function at three
years as well as lower rates of lung transplant and death in people
with cystic fibrosis, compared to pre-TRIKAFTA® initiation -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor
and ivacaftor), also known in the European Union and in the U.K. as
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with
ivacaftor, were presented at this year’s European Cystic Fibrosis
Society’s (ECFS) 47th European Cystic Fibrosis Conference held June
5-8, 2024, in Glasgow, Scotland.
Data from a randomized, double-blind, Phase 3 study (abstract
WS06.04) demonstrated that people with CF who have rare,
non-F508del mutations in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene responsive to TRIKAFTA® in vitro
demonstrated clinical benefit from receiving TRIKAFTA®. Compared to
placebo, lung function improved by 9.2 percentage points as
measured by ppFEV1, CFTR function improved (as measured by mean
sweat chloride concentration reductions of 28.3 mmol/L), and
pulmonary exacerbations were reduced by 72% per year. Safety and
tolerability were generally consistent with the established safety
profile of TRIKAFTA®.
Vertex also presented the interim analysis (IA) of a
registry-based study of real-world data collected from people with
CF initiating TRIKAFTA® from 2019-20 in the U.S. and KAFTRIO® plus
ivacaftor from 2020-21 in Germany (abstract WS01.04). The ongoing
five-year post-authorization study is the largest real-world study
of people with CF treated with TRIKAFTA®/KAFTRIO® to date,
including more than 16,000 people with CF from the U.S. Cystic
Fibrosis Foundation Patient Registry (CFFPR) and approximately
3,000 people with CF from the German CF Registry. The IA showed
clinically meaningful, disease-modifying benefits for
TRIKAFTA®/KAFTRIO®, including a 76% and 70% reduction in the
cumulative annual rate of pulmonary exacerbations in the U.S. and
in Germany, respectively, compared to the year prior to
TRIKAFTA®/KATRIO® treatment. In addition, there was a 62% lower
rate of death in the U.S. and 84% lower in Germany; and an 86%
lower rate of lung transplant in the U.S. and 96% lower in Germany
compared to the 2019 U.S. CFFPR and German CF Registry populations
(pre-TRIKAFTA®/KAFTRIO®). No new safety concerns were
identified.
“The breadth of TRIKAFTA data presented at ECFS is further
evidence of the significant potential of this disease-modifying
medicine,” said Carmen Bozic, M.D., Executive Vice President,
Global Medicines Development and Medical Affairs, and Chief Medical
Officer at Vertex. “These studies demonstrate that TRIKAFTA is
changing the course of CF treatment and the lives of those living
with CF.”
Additional Presentations
Other Vertex presentations at the conference this year
include:
- Abstract WS15.02, entitled “Real-World Effectiveness of
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in People With
Cystic Fibrosis and ELX/TEZ/IVA-Responsive, Non-F508del CFTR
Genotypes”
- Abstract EPS10.08, entitled “LONGITUDE: An Observational Study
of the Long-term Effectiveness of ELX/TEZ/IVA in People With CF
Using Data From the UK CF Registry – Preliminary Results From the
Subgroup Aged 6-11 Years”
- Abstract P096, entitled “Qualitative Interviews Confirming the
Faithful Electronic Migration of the Preschool Pictorial Cystic
Fibrosis Questionnaire-Revised (CFQ-R) and Parent Preschool
CFQ-R”
- Abstract EPS6.05, entitled “Clinical Outcomes in Concurrent
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Treated vs.
Ineligible Cohorts in the US Cystic Fibrosis Foundation Patient
Registry (CFFPR) During COVID-19”
- Abstract P063, entitled “ELX/TEZ/IVA has beneficial effects on
clinical outcomes and quality of life in people with cystic
fibrosis in the real-world TRAJECTORY study”
- Abstract P072, entitled “Real-World Impact of
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Italy: A
Retrospective Study From a CF Center”
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 92,000 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.
Today Vertex CF medicines are treating over 65,000 people with
CF across 60 countries on six continents. This represents 2/3 of
the diagnosed people with CF eligible for CFTR modulator
therapy.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
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. TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and 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. 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
elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus
from the airways.
TRIKAFTA U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION
FOR 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.
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 and in the U.K. 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 and
on https://products.mhra.gov.uk/.
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 acute and neuropathic
pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal
dominant polycystic kidney disease, type 1 diabetes, myotonic
dystrophy type 1 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, Latin America and the
Middle East. 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 statements regarding our
expectations for the benefits of TRIKAFTA/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 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 studies may not be indicative of final clinical trial
results, 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, that data may not be available
on the anticipated timeline, or at all, that our development
programs may experience delays, and other risks listed under the
heading “Risk Factors” in Vertex's most recent annual report and
subsequent filings 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)
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