- With this approval approximately 1,500
children with one minimal function mutation and one F508del
mutation have a medicine to treat the underlying cause of their
disease for the first time -
First paragraph, third sentence of release should read: An
additional dosage strength of TRIKAFTA tablets is now available
(elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor
75 mg)...(instead of An additional dosage strength of TRIKAFTA
tablets is now available (elexacaftor 50 mg/tezacaftor 25
mg/ivacaftor 75 mg and ivacaftor 75 mg)...).
The updated release reads:
VERTEX ANNOUNCES U.S. FDA APPROVAL FOR
TRIKAFTA® (ELEXACAFTOR/TEZACAFTOR/IVACAFTOR AND IVACAFTOR) IN
CHILDREN WITH CYSTIC FIBROSIS AGES 6 THROUGH 11 WITH CERTAIN
MUTATIONS
- With this approval approximately 1,500
children with one minimal function mutation and one F508del
mutation have a medicine to treat the underlying cause of their
disease for the first time -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the U.S. Food and Drug Administration (FDA) approved
expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) to include children with cystic fibrosis (CF) ages 6
through 11 years who have at least one F508del mutation in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene or
a mutation in the CFTR gene that is responsive to TRIKAFTA based on
in vitro data. TRIKAFTA was previously approved by the FDA for use
in people with cystic fibrosis 12 years and older with at least one
copy of the F508del mutation or one copy of a mutation that is
responsive in vitro. An additional dosage strength of TRIKAFTA
tablets is now available (elexacaftor 50 mg/tezacaftor 25
mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this
approval.
“Today’s approval is a critical milestone in our efforts to
deliver medicines that help treat the underlying cause of this
devastating disease as early in life as possible,” said Reshma
Kewalramani, M.D., Chief Executive Officer and President, Vertex.
“We can now reach approximately 1,500 newly eligible children in
the U.S., and we continue to pursue approval for this expanded
indication in other countries.”
Vertex completed a 24-week Phase 3 open-label, multicenter study
which enrolled 66 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
to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA.
The regimen was generally well tolerated, and safety data were
similar with those observed in previous studies of patients ages 12
years and older. The full data from this study were recently
published in American Journal of Respiratory and Critical Care
Medicine.
“Clinical experience with TRIKAFTA in patients 12 and older over
the past 20 months has demonstrated this medicine has a meaningful
and unprecedented clinical benefit for patients. I look forward to
now being able to treat younger patients with this breakthrough
medicine, including those who have not presented major signals of
disease progression,” said Terri Laguna M.D., M.S.C.S., Associate
Director of the Cystic Fibrosis Center and Division Head, Pulmonary
and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital
of Chicago. “In addition to bringing TRIKAFTA to a younger patient
population, patients not previously eligible for any CFTR modulator
will now be able to access a treatment that targets the underlying
cause of their disease.”
TRIKAFTA® is already approved for the treatment of patients with
CF ages 12 years and older with certain mutations in the U.S.
Switzerland, Australia and Israel, as well as in the EU and the
U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
combination regimen with KALYDECO® (ivacaftor). Vertex has
submitted applications for use of TRIKAFTA/KAFTRIO in children ages
6 through 11 years to the European Medicines Agency (EMA) and the
Medicines & Healthcare products Regulatory Agency (MHRA) and
plans to file for this expanded use in Switzerland, Australia and
Israel this year.
For more information on TRIKAFTA, patient assistance programs or
to find additional eligibility details, visit TRIKAFTA.com,
VertexGPS.com or vertextreatments.com.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 80,000 people globally. 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.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS
What is TRIKAFTA?
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 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 6 years of age.
Patients should not take TRIKAFTA if they take certain
medicines or herbal supplements, such as: antibiotics such as
rifampin or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; or 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.
TRIKAFTA may cause dizziness in some people who take it.
Patients should not drive a car, operate machinery, or do anything
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) has happened 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.
These are not all the possible side effects of TRIKAFTA.
Please click the product link 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 cell and genetic 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. 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 11 consecutive years on Science magazine's Top
Employers list and a best place to work for LGBTQ equality by the
Human Rights Campaign. 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. Reshma
Kewalramani and Dr. Terri Laguna in this press release, statements
regarding our expectations for the number of patients newly
eligible for TRIKAFTA, and statements regarding the potential
benefits of TRIKAFTA and our expectations regarding global
regulatory filings for the expanded use of TRIKAFTA. 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 our regulatory
filings may not be approved on a timely basis, or at all, 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, and other risks listed under the heading
“Risk Factors” in Vertex's most recent annual report 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. 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 or 617-961-7163
Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
International: +44 20 3204 5275
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