Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the company has completed a global Phase 3 study of
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in
children ages 6 through 11 years old with cystic fibrosis (CF) who
have either two copies of the F508del mutation or one copy of the
F508del mutation and one minimal function mutation, and based on
the results will submit a supplemental New Drug Application (sNDA)
to the U.S. Food and Drug Administration (FDA) in the fourth
quarter of 2020, with additional global regulatory submissions to
follow.
“Our aim is to extend eligibility to all patients who may
benefit from this transformative medicine, and the positive results
from the study in children ages 6 through 11 years old allows us to
take another step forward toward this goal,” said Carmen Bozic,
M.D., Executive Vice President, Global Medicines Development and
Medical Affairs, and Chief Medical Officer at Vertex. “We are
looking forward to filing an sNDA in the coming months and bringing
TRIKAFTA to younger people with CF.”
Bringing TRIKAFTA to Children Less than 12 Years of
Age
The 24-week global Phase 3 open-label study evaluated the safety
and efficacy of TRIKAFTA in 66 children ages 6 through 11 years old
who have either two copies of the F508del mutation or one copy of
the F508del mutation and one minimal function mutation. The primary
endpoint of the study was safety and tolerability, and the results
showed that TRIKAFTA was generally well tolerated and the safety
data were consistent with those observed in previous Phase 3
studies. In addition, clinically meaningful improvements were seen
across multiple secondary efficacy endpoints, including
improvements in percent predicted forced expiratory volume in 1
second (ppFEV1), sweat chloride, Cystic Fibrosis Questionnaire
Revised (CFQ-R) respiratory domain score, body mass index (BMI) and
other measures through 24 weeks of treatment. The study showed the
benefit-risk profile of TRIKAFTA in children with CF ages 6 through
11 years old was similar to that seen in people with CF ages 12 and
older in the Phase 3 studies which have supported approval. Based
on the results, Vertex will submit an sNDA to the U.S. FDA in the
fourth quarter of 2020, with additional global regulatory
submissions to follow.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting approximately 75,000 people worldwide. CF is a
progressive, multi-system disease that affects the lungs, liver, GI
tract, sinuses, sweat glands, pancreas 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. While
there are many different types of CFTR mutations that can cause the
disease, the vast majority of all people with CF have at least one
F508del mutation. These mutations, which can be determined by a
genetic test, or genotyping test, lead to CF by creating
non-working and/or too few CFTR proteins 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 that can cause chronic lung infections and
progressive lung damage in many patients that eventually leads to
death. The median age of death is in the early 30s.
About TRIKAFTA®
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a
prescription medicine used for the treatment of cystic fibrosis
(CF) in patients ages 12 years and older who have at least one copy
of the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (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 12
years of age. TRIKAFTA is designed to increase the quantity and
function of the F508del-CFTR protein at the cell surface. The
approval of TRIKAFTA was supported by positive results of two
global Phase 3 studies in people ages 12 years and older with CF: a
24-week Phase 3 study in 403 people with one F508del mutation and
one minimal function mutation (F/MF) and a 4-week Phase 3 study in
107 people with two F508del mutations (F/F).
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TABLETS
TRIKAFTA is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 12 years and older who have
at least one copy of the F508del mutation in the cystic fibrosis
transmembrane conductance regulator (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 12 years of age.
Patients should not take TRIKAFTA if they take certain
medicines, such as: antibiotics such as rifampin or rifabutin;
seizure medicines such as phenobarbital, carbamazepine, or
phenytoin; St. John’s wort.
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.
TRIKAFTA may affect the way other medicines work, and other
medicines may affect how TRIKAFTA works. Therefore, the dose of
TRIKAFTA may need to be adjusted when taken with certain medicines.
Patients should especially tell their doctor if they take:
antifungal medicines including ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; antibiotics including
telithromycin, clarithromycin, or erythromycin; other medicines
including rifampin, rifabutin, phenobarbital, carbamazepine,
phenytoin, and St. John’s wort.
TRIKAFTA may cause dizziness in some people who take it.
Patients should not drive a car, operate machinery, or do anything
that requires alertness until they know how TRIKAFTA affects
them.
Patients should avoid food or drink that contains
grapefruit while they are taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
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) 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, 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 TRIKAFTA.
Please click here to see the full Prescribing Information for
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 medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney diseases. In addition, Vertex has a rapidly
expanding pipeline of genetic and cell therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
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, UK. 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 10 consecutive years on Science magazine's Top
Employers list and top five on the 2019 Best Employers for
Diversity list by Forbes. 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,
including, without limitation, statements made by Dr. Carmen Bozic
in this press release, statements regarding the potential benefits
of TRIKAFTA, our plans to make additional submissions to the FDA
and other global regulatory agencies, including the timing of
additional regulatory submissions, and our expectations regarding
additional regulatory reviews and approvals of our medicines. 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
regulatory authorities may not approve, or approve on a timely
basis, the sNDA, 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 Risk Factors in Vertex's annual report and subsequent
quarterly reports filed with the Securities and Exchange Commission
and available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200910005580/en/
Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com or 617-961-7163
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
International: +44 20 3204 5275
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Apr 2024 to May 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From May 2023 to May 2024