– Phase 3 data on investigational vanza triple
demonstrates non-inferiority to TRIKAFTA® in ppFEV1 and further
improvement of CFTR function as measured by sweat chloride –
– Real-world evidence and clinical studies of
TRIKAFTA® continue to show sustained long-term benefits including
improvement in pancreatic function in young patients –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the first accepted medical presentations of the Phase 3
data on the investigational once daily
vanzacaftor/tezacaftor/deutivacaftor (“vanza triple”) — the
potential next-in-class triple combination medicine — will take
place at the North American Cystic Fibrosis Conference (NACFC).
Vertex also announced presentations describing long-term outcomes
in people with cystic fibrosis (CF) ages 2 to 11 years taking
TRIKAFTA®, demonstrating consistent and sustained improvements
across multiple measures of disease.
“TRIKAFTA has transformed the treatment of CF in the 5 years
since its approval, changing the outlook for patients with CF,”
said Carmen Bozic, M.D., Executive Vice President, Global Medicines
Development and Medical Affairs, and Chief Medical Officer at
Vertex. “Toward our goal to bring all eligible patients to normal
levels of CFTR function, we have made global regulatory submissions
for our next-in-class vanza triple combination medicine for
patients with CF aged 6 years and older. We are excited to bring
this promising new medicine, which has the potential to deliver
even greater improvements in sweat chloride, to patients with
CF.”
Data on the Investigational Once Daily Next-in-Class Triple
Combination Therapy, vanzacaftor/tezacaftor/deutivacaftor
Vertex will present data on the vanza triple in an oral
presentation and two posters on Friday, September 27. This is the
first time that the clinical data from the Phase 3 clinical trials
of the vanza triple in patients 6 years and older with CF have been
accepted for presentation at a medical meeting. These data formed
the basis of global regulatory submissions.
“The additional, significant reductions in sweat chloride we see
in the vanza triple clinical trials are noteworthy. I believe this
improvement in CFTR function may lead to important benefits for
people with CF,” said Claire L. Keating, M.D., Co-Director of the
Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia
University. “If approved, as a clinician, I’m looking forward to
being able to offer patients an option, with once daily dosing,
that could advance the treatment for people living with CF.”
Data from the Phase 3 clinical studies of the vanza triple will
be presented in the following sessions:
- “Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in
Adolescents and Adults with CF: Results from Two Randomized,
Active-Controlled Phase 3 Trials,” will be an oral presentation
during a session entitled: “Cutting Edge Research: What’s New” on
Friday, September 27, from 10:15 a.m. to 12:15 p.m. EDT and be a
poster presentation on Friday, September 27, from 1:15-2:15 p.m.
EDT.
- “Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor
(VNZ Triple) in Children 6 Through 11 Years of Age with Cystic
Fibrosis,” will be a poster presentation on Friday, September 27,
from 1:15-2:15 p.m. EDT.
The vanza triple was granted Fast Track and Orphan Drug
Designations from the U.S. Food and Drug Administration (FDA) for
the treatment of CF and has been submitted for regulatory approval
in the U.S., Canada, U.K., EU, Switzerland, Australia and New
Zealand. Vertex has been assigned a Prescription Drug User Fee Act
(PDUFA) date by the FDA of January 2, 2025, for this submission.
The vanza triple has not been approved by any global health
authority.
Long-Term Benefits of TRIKAFTA®
Vertex will present new data on TRIKAFTA® from long-term (96
week and 192 week) studies in patients ages 2-11 years old that
reinforce the sustained benefit seen in studies in older people
with CF. Specifically, that early treatment with TRIKAFTA® is
associated with sustained improvements in lung function. These new
data demonstrate that in these young children, TRIKAFTA® could also
lead to improved exocrine pancreatic function over time. The data
presented at NACFC highlight the safety and tolerability of
TRIKAFTA®, which were generally consistent with the established
safety profile.
“I have seen first-hand the positive long-term impact that
improvement of CFTR function by TRIKAFTA can have on patients’
clinical outcomes. These improvements are particularly striking for
me as a physician caring for young children, where improvements in
things like lung function, pancreatic function and quality of life
are so meaningful,” said Professor Marcus A. Mall, M.D., Professor
and Chair of the Department of Pediatric Respiratory Medicine,
Immunology and Critical Care Medicine and Cystic Fibrosis Center at
Charité - Universitätsmedizin Berlin.
Vertex will have four poster presentations that include clinical
trial and real-world evidence data, three specifically on TRIKAFTA®
and one showing sustained benefits from KALYDECO®, which is
approved for treatment in the youngest ages.
- “Long-term safety and efficacy of
elexacaftor/tezacaftor/ivacaftor in children 2 years and older with
cystic fibrosis and at least one F508del allele: 96-week results
from an open-label extension study,” will be a poster presentation
on Friday, September 27, at 12:15-1:15 p.m. EDT.
- “Long-term safety and efficacy of
elexacaftor/tezacaftor/ivacaftor in children 6 years and older with
cystic fibrosis and at least one F508del allele: Results from a
192-week extension study,” will be a poster presentation on Friday,
September 27, at 1:15-2:15 p.m. EDT.
- “Effectiveness of elexacaftor/tezacaftor/ivacaftor
(ELX/TEZ/IVA) in people with cystic fibrosis and non-F508del CFTR
variants: Interim results from a registry-based study,” will be a
poster presentation on Friday, September 27, from 12:15-1:15 p.m.
EDT.
- “Long-term benefits of early ivacaftor (IVA) initiation in
people with cystic fibrosis (CF),” will be a poster presentation on
Friday, September 27, from 1:15-2:15 p.m. EDT.
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.
Learn more about the importance of sweat chloride (SwCl) in
cystic fibrosis.
Today Vertex CF medicines are treating over 68,000 people with
CF across more than 60 countries on six continents. This represents
2/3 of the diagnosed people with CF eligible for CFTR modulator
therapy.
About vanzacaftor/tezacaftor/deutivacaftor (the “vanza
triple”)
In people with CF, mutations in the CFTR gene lead to decreased
quantity and/or function of the CFTR protein channel at the cell
surface. Vanzacaftor and tezacaftor are designed to increase the
amount of CFTR protein at the cell surface by facilitating the
processing and trafficking of the CFTR protein. Deutivacaftor is a
potentiator designed to increase the channel open probability of
the CFTR protein delivered to the cell surface to improve the flow
of salt and water across the cell membrane.
Investigational vanzacaftor/tezacaftor/deutivacaftor was granted
Fast Track and Orphan Drug Designations from the U.S. Food and Drug
Administration for the treatment of CF. The vanza triple will be
subject to a meaningfully lower single-digit royalty obligation,
compared to the rate payable on Vertex’s current CF portfolio.
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® (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, 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 2 years
of age.
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.
KALYDECO® U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION
FOR INDICATIONS AND USAGE
INDICATIONS AND USAGE
KALYDECO (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients age 1 month and older
who have at least one mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) 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 1 month of age.
IMPORTANT SAFETY INFORMATION
Before taking KALYDECO, patients should tell their doctor
about all their medical conditions, including if they: have
liver or kidney problems; are allergic to KALYDECO or any
ingredients; are pregnant or plan to become pregnant because it is
not known if KALYDECO will harm an unborn baby; and are
breastfeeding or planning to breastfeed because is not known if
KALYDECO 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. KALYDECO may affect
the way other medicines work, and other medicines may affect
how KALYDECO works. 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 the antibiotics rifampin
or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; St. John’s wort; antifungal medicines
such as ketoconazole, itraconazole, posaconazole, voriconazole, or
fluconazole; or antibiotics such as telithromycin, clarithromycin,
or erythromycin.
KALYDECO can cause dizziness in some patients who take
it. If patients experience dizziness, they should not drive or
operate machines until symptoms improve.
Patients should avoid food or drink containing grapefruit
while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood, which have happened in
patients receiving KALYDECO. The patient’s doctor will do
blood tests to check their liver before starting KALYDECO, every 3
months during the first year of taking KALYDECO, and every year
while taking KALYDECO. 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.
Serious allergic reactions have happened to patients who
are treated with KALYDECO. Patients should call their healthcare
provider or go to the emergency room right away if they have
symptoms of an allergic reaction. Symptoms of an allergic reaction
may include rash or hives, tightness of the chest or throat or
difficulty breathing, and light-headedness or dizziness.
Abnormality of the eye lens (cataract), which has
happened in some children and adolescents receiving KALYDECO. The
patient’s doctor should perform eye examinations before and during
treatment with KALYDECO to look for cataracts.
The most common side effects 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.
Use of KALYDECO in patients aged 1 month to less than 6 months
born from a pregnancy lasting (gestational age) less than 37 weeks
has not been evaluated.
These are not all the possible side effects of KALYDECO.
Please click here to see the full Prescribing Information
for KALYDECO.
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, the statements by Carmen
Bozic, M.D., Professor Marcus A. Mall, M.D., and Claire L. Keating,
M.D., in this press release, and statements regarding our
expectations for and the anticipated benefits of the vanza triple,
our expectations that medical presentations of Phase 3 data on the
vanza triple will take place at NACFC, our expectations for the
long-term data for TRIKAFTA, plans to present long-term TRIKAFTA
data and KALYDECO data, and our expectations for a lower royalty
obligation for the vanza triple. While we believe 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 or further
development of its compounds due to safety, efficacy, and other
reasons, 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)
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Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
Heather Nichols: +1 617-839-3607 or International: +44 20 3204
5275
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