- TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor
and ivacaftor) real-world safety and effectiveness interim results
show improved lung function and significant reductions in risk of
pulmonary exacerbations, lung transplant and death for people with
cystic fibrosis (CF) -
- Study in people with CF (F/F or F/MF
genotypes) treated with TRIKAFTA® shows no mean loss of lung
function after two years compared to those not treated with a CFTR
modulator -
- Long-term real-world study results show
significant benefits of initiating KALYDECO® (ivacaftor) at young
age -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that five scientific abstracts on the company’s portfolio
of cystic fibrosis (CF) medicines will be presented at the European
Cystic Fibrosis Society's (ECFS) 45th European Cystic Fibrosis
Conference held June 8-11, 2022, in Rotterdam, the Netherlands.
Vertex will present the first analysis of data collected in the
U.S. CF Foundation Patient Registry (CFFPR) of over 16,000 people
with CF treated with TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor
and ivacaftor) for an average of nine months. This first interim
analysis of an ongoing five-year post-authorization study (abstract
WS22.05) showed that real-world treatment with TRIKAFTA® was
associated with improved lung function and a 77% reduced risk of
pulmonary exacerbations compared to pre-TRIKAFTA® baseline, as well
as an 87% lower risk of lung transplant and a 74% lower risk of
death, compared to the historical 2019 U.S. CFFPR population. No
new safety concerns were identified.
Vertex will also present data comparing the annual rate of lung
function change in people with CF ages 12 years and older with two
F508del mutations (F/F) or one F508del mutation and one minimal
function mutation (F/MF) treated with TRIKAFTA® in pivotal studies
and an open-label extension study compared to propensity-score
matched historical CFTR-modulator-untreated controls from the U.S.
CFFPR (abstract WS22.04). Results show that TRIKAFTA® demonstrated
on average no decrease in ppFEV1 over a two-year period in this
population, in contrast to declines seen in the matched controls.
The analysis indicates that treatment with TRIKAFTA® has a
significant impact on the trajectory of CF lung disease.
Additionally, Vertex will present data from a long-term
real-world study demonstrating that initiating KALYDECO®
(ivacaftor) early in life (ages 6-10 years) preserves lung function
to a greater extent than if KALYDECO® is initiated at an older age
(abstract WS17.03). These results show the importance of early
initiation of KALYDECO® for eligible patients.
“These long-term and real-world studies show the potentially
transformative benefits of treatment with CFTR modulators and add
to the substantial body of evidence supporting treatment as early
in life as possible,” said Carmen Bozic, M.D., Executive Vice
President, Global Medicines Development and Medical Affairs, and
Chief Medical Officer at Vertex. “We continue to make rapid
progress in developing medicines that treat the underlying cause of
CF, and today, we are closer to our goal of developing highly
effective therapies for all patients with CF than ever before.”
Additional Presentations
In addition to the studies noted above, other Vertex
presentations at the conference this year support the long-term and
early use of CFTR modulators:
- Abstract WS08.04 — Results of real-world study in people with
CF with select residual function mutations, treated with KALYDECO®
(ivacaftor)
- Abstract WS17.02 — Results from an ORKAMBI®
(lumacaftor/ivacaftor) exploratory phase 2 open-label extension
study in children with CF ages 2-5
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 83,000 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 the 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 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 elexacaftor,
tezacaftor and ivacaftor help hydrate and clear mucus from the
airways.
TRIKAFTA® is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 6 years and older who have at
least one copy of the F508del mutation, or another mutation
responsive to TRIKAFTA®, in the CFTR gene. 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 6 years of age.
Please see Important Safety Information below and [click here]
for full U.S. Prescribing Information.
About KALYDECO® (ivacaftor)
In people with certain types of mutations in the CFTR gene, the
CFTR protein at the cell surface does not function properly. Known
as a CFTR potentiator, ivacaftor is an oral medicine designed to
facilitate the ability of CFTR proteins to transport salt and water
across the cell membrane, which helps hydrate and clear mucus from
the airways. KALYDECO® (ivacaftor) was the first medicine to treat
the underlying cause of cystic fibrosis (CF) in people with
specific mutations in the CFTR gene.
KALYDECO® is a prescription medicine used for the treatment of
CF in patients aged 4 months and older who have at least one
mutation in their CF gene that is responsive to KALYDECO®. Patients
should talk to their doctor to learn if they have an indicated CF
gene mutation. It is not known if KALYDECO® is safe and effective
in children under 4 months of age.
Please see Important Safety Information below and [click here]
for full U.S. Prescribing Information.
About ORKAMBI® (lumacaftor/ivacaftor)
In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little to no CFTR protein at the cell surface.
ORKAMBI® (lumacaftor/ivacaftor) is an oral medicine that is a
combination of lumacaftor and ivacaftor. Lumacaftor is designed to
increase the amount of mature protein at the cell surface by
targeting the processing and trafficking defect of the F508del-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
lumacaftor and ivacaftor help hydrate and clear mucus from the
airways.
ORKAMBI® is a prescription medicine used for the treatment of CF
in patients age 2 years and older who have two copies of the
F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI®
should only be used in these patients. It is not known if ORKAMBI®
is safe and effective in patients under 2 years of age.
Please see Important Safety Information below and [click here]
for full U.S. Prescribing Information.
IMPORTANT SAFETY INFORMATION for TRIKAFTA
(elexacaftor/tezacaftor/ivacaftor and ivacaftor), KALYDECO
(ivacaftor), and ORKAMBI (lumacaftor/ivacaftor)
Patients should not take KALYDECO or TRIKAFTA if they take
certain medicines or herbal supplements, such as: the
antibiotics rifampin or rifabutin; seizure medicines such as
phenobarbital, carbamazepine, or phenytoin; or St. John’s wort.
Patients should not take ORKAMBI if they take certain
medicines or herbal supplements, such as: the antibiotics
rifampin or rifabutin; the seizure medicines phenobarbital,
carbamazepine, or phenytoin; the sedatives and anti-anxiety
medicines triazolam or midazolam; the immunosuppressant medicines
cyclosporine, everolimus, sirolimus, or tacrolimus; or St. John’s
wort.
Before taking KALYDECO, ORKAMBI, or TRIKAFTA patients should
tell their doctor about all of their medical conditions,
including if they: have or have had liver problems; have kidney
problems; are pregnant or plan to become pregnant because it is not
known if KALYDECO, ORKAMBI, or TRIKAFTA, will harm an unborn baby;
or are breastfeeding or planning to breastfeed because it is not
known if KALYDECO, ORKAMBI, or TRIKAFTA passes into breast milk.
Before taking ORKAMBI, patients should tell their doctor if they
have had an organ transplant, or if they are using a hormonal
contraceptive including oral, injectable, transdermal, or
implantable form as this should not be used as a method of birth
control when taking ORKAMBI.
KALYDECO, ORKAMBI, or TRIKAFTA may affect the way other
medicines work, and other medicines may affect how KALYDECO,
ORKAMBI, or TRIKAFTA work. Therefore, the dose of KALYDECO,
ORKAMBI, or TRIKAFTA may need to be adjusted when taken with
certain medications. Patients should especially tell their doctor
if they take antifungal medications such as ketoconazole,
itraconazole, posaconazole, voriconazole, or fluconazole; or
antibiotics such as telithromycin, clarithromycin, or
erythromycin.
KALYDECO or TRIKAFTA can cause dizziness in some people
who take it. Patients should not drive a car, use machinery, or do
anything that needs them to be alert until they know how KALYDECO
or TRIKAFTA affects them.
When taking ORKAMBI, patients should tell their doctor if
they stop taking ORKAMBI for more than 1 week as their doctor
may need to change the dose of ORKAMBI or other medicines the
patient is taking.
Patients should avoid food or drink containing grapefruit
while taking KALYDECO or TRIKAFTA.
KALYDECO, ORKAMBI, and TRIKAFTA can cause serious side
effects, such as:
Liver damage and worsening of liver function in people taking
TRIKAFTA 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 have been reported in
patients receiving KALYDECO, ORKAMBI, or TRIKAFTA. The
patient's doctor will do blood tests to check their liver before
starting treatment with KALYDECO, ORKAMBI, or TRIKAFTA; every 3
months during the first year of treatment; and every year while on
treatment. For patients who have had high liver enzymes in the
past, the doctor may do blood tests to check the liver more often.
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 their skin or
the white part of their eyes; loss of appetite; nausea or vomiting;
or dark, amber colored urine.
Worsening of liver function in people with severe liver
disease taking ORKAMBI. The worsening of liver function can be
serious or cause death. Talk to your doctor if you have been told
you have liver disease as your doctor may need to adjust the dose
of ORKAMBI.
Breathing problems such as shortness of breath or chest
tightness in patients when starting ORKAMBI, especially in
patients who have poor lung function. If a patient has poor lung
function, their doctor may monitor them more closely when starting
ORKAMBI.
An increase in blood pressure in some people receiving
ORKAMBI. The patient’s doctor should monitor their blood
pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) in some children and
adolescents treated with KALYDECO, ORKAMBI, or TRIKAFTA. If the
patient is a child or adolescent, their doctor should perform eye
examinations before and during treatment with KALYDECO, ORKAMBI, or
TRIKAFTA to look for cataracts.
The most common side effects of KALYDECO include
headache; upper respiratory tract infection (common cold), which
includes sore throat, nasal or sinus congestion, and runny nose;
stomach (abdominal) pain; diarrhea; rash; nausea; and
dizziness.
The most common side effects of ORKAMBI include breathing
problems, such as shortness of breath and chest tightness; nausea;
diarrhea; fatigue; increase in a certain blood enzyme called
creatinine phosphokinase; rash; gas; common cold, including sore
throat, stuffy or runny nose; flu or flu-like symptoms; and
irregular, missed, or abnormal periods (menses) and increase in the
amount of menstrual bleeding. Additional side effects seen in
children include cough with sputum, stuffy nose, headache, stomach
pain, and increase in sputum.
The most common side effects of TRIKAFTA include
headache; diarrhea; upper respiratory tract infection (common
cold), including stuffy and runny nose; stomach (abdominal) pain;
inflamed sinuses; increase in liver enzymes; increase in a certain
blood enzyme called creatine phosphokinase; rash; flu (influenza);
and increase in blood bilirubin.
These are not all the possible side effects of KALYDECO,
ORKAMBI, or TRIKAFTA. Please click product link to see the full
U.S. Prescribing Information for KALYDECO,
ORKAMBI, or TRIKAFTA.
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
pipeline of investigational small molecule, cell and genetic
therapies in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes,
alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
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 12 consecutive years on Science magazine's Top
Employers list and one of the 2021 Seramount (formerly Working
Mother Media) 100 Best Companies. 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 Dr.
Bozic in this press release, statements regarding the potential
benefits, safety and efficacy of our products, and our plans to
present data about our portfolio of CF products at the ECFS
European Cystic Fibrosis Conference, including an analysis of data
from the ongoing five-year post-authorization safety study for
TRIKAFTA®, data comparing the annual rate of lung function change
in certain individuals with CF and our assessment of the impact of
such data, data regarding the early initiation of KALYDECO® and our
assessment of the impact of such data, and additional scientific
presentations regarding our marketed CF products, including
expectations regarding the abstracts that will be made available 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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Vertex Pharmaceuticals Incorporated Investors: Michael
Partridge, +1 617-341-6108 or Manisha Pai, +1 617-961-1899
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or
International: +44 20 3204 5275
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