-Interim results of largest real-world study of
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed
sustained improvement in lung function, reduction in pulmonary
exacerbations frequency and lower rates of lung transplant and
death for people with cystic fibrosis-
- Twelve presentations add to the body of
evidence supporting the use of CFTR modulators for all eligible
people with cystic fibrosis-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that 12 scientific abstracts on the company’s portfolio
of cystic fibrosis (CF) medicines were presented at this year’s
European Cystic Fibrosis Society's (ECFS) 46th European Cystic
Fibrosis Conference held June 7-10, 2023, in Vienna, Austria.
Together, the data presented show the long-term benefits of
treatment with CFTR modulators as well as the importance of
treating the underlying cause of CF as early in life as possible.
Key data presented at this year’s conference are highlighted
below.
Vertex presented an interim analysis (IA) of a registry-based
study of real-world data collected from people with CF and treated
with 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,
including over 16,000 people with CF from the Cystic Fibrosis
Foundation Patient Registry (CFFPR) and nearly 3,000 from the
German CF Registry. This ongoing five-year post-authorization study
(abstract WS16.03) is the largest real-world study of people with
CF treated with TRIKAFTA® to date. The IA showed clinically
significant disease-modifying benefits for TRIKAFTA®, including
improved lung function and a 79% reduction of pulmonary
exacerbations in the U.S. and 83% in Germany overall compared to
pre-TRIKAFTA® baseline. The rate of death was 72% lower in the U.S.
and 82% lower in Germany, the rate of lung transplant was 85% lower
in the U.S. and 100% lower in Germany, compared to 2019
(pre-TRIKAFTA®) U.S. CFFPR and German CF Registry populations. No
new safety concerns were identified.
Vertex also presented final results of the nearly four-year
TRIKAFTA® open-label follow-up study of the Phase 3 pivotal trials
in people with CF ages 12 years and older with at least one F508del
mutation in the CFTR gene (Late Breaking Science; Workshop 15). The
results of this study are unprecedented, showing for the first time
that treatment with TRIKAFTA® resulted in no decline in lung
function over a four-year period.
“CFTR modulators markedly improve clinical outcomes of people
living with CF as demonstrated in the large and growing quantity of
data presented at ECFS this year,” said Professor Isabelle Fajac,
Professor of Physiology, Cochin Hospital, Assistance
Publique-Hôpitaux de Paris, Université Paris Cité, Paris, France.
“The data on TRIKAFTA in particular demonstrate that this medicine
improves lung function sustainably and in a real-world setting. It
also reduces the risks of pulmonary exacerbation, death and lung
transplant, and it is generally well tolerated.”
Additional Presentations
Other Vertex presentations at the conference this year
include:
- Abstract EPS6.05 entitled “A Phase 3b study of the effects of
elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and
physical activity in people with cystic fibrosis (CF)”
- Abstract WS05.04 entitled “Safety and efficacy of ivacaftor
(IVA) in children aged 1 to <4 months with cystic fibrosis
assessed with an innovative clinical trial design”
- Late Breaking Science (Workshop 15) abstract entitled
“LONGITUDE: An observational study of the long-term effectiveness
of ivacaftor/tezacaftor/elexacaftor in people with cystic fibrosis
using data from the United Kingdom Cystic Fibrosis Registry”
- Abstract WS16.02 entitled “Real-world (RW) clinical
effectiveness of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in
children with cystic fibrosis aged 6-11 years: interim results from
the HELIO study”
- Abstract P117 entitled “A longitudinal study on the impact of
ELX/TEZ/IVA treatment on quality of life in people with cystic
fibrosis in the real world”
- Abstract P118 entitled “Real-world impact of ELX/TEZ/IVA on
quality of life of children with CF aged 6-11 years and primary
caregivers in the UK: MAGNIFY, a prospective, observational,
noninterventional study”
- Abstract WS05.03 entitled “A Phase 3b study of the effects of
elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on glucose tolerance
in people with cystic fibrosis (CF) and abnormal glucose
metabolism”
- Abstract WS16.04 entitled “Benefits of lumacaftor/ivacaftor
(LUM/IVA) initiation in children with CF aged 2 through 5 years:
Interim results from an ongoing registry-based study”
- Abstract WS16.05 entitled “Long-Term Impact of Ivacaftor (IVA)
in People with Cystic Fibrosis in Ireland”
- Late Breaking Science (Workshop 15) abstract entitled “Effects
of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment on
markers of inflammation in people with cystic fibrosis (CF)”
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 early 30s.
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.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TRIKAFTA 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.
Before taking TRIKAFTA, patients should tell their doctor
about all of their medical conditions, including if they: 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.
Tell your doctor about all the medicines you 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 they are taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in people
with severe liver disease that can be serious and may require
transplantation. Liver damage has also happened in people without
liver disease.
High liver enzymes in the blood, which is a common side
effect in people 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.
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 small molecule, mRNA, cell and
genetic therapies (including gene editing) 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.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is 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 13 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 Facebook, Twitter,
LinkedIn, YouTube and Instagram.
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 made by
Professor Isabelle Fajac in this press release, and statements
regarding the potential benefits, safety and efficacy of our
medicines, and our plans to present data about our medicines at the
ECFS European Cystic Fibrosis Conference. 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
development programs may not support registration, approval or
further development of its compounds due to safety, efficacy or
other reasons, risks related to approval and commercialization of
our medicines, 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
(SEC) and available through the company's website at www.vrtx.com
and on the SEC’s website at www.sec.gov. 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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