-About 900 children with cystic fibrosis will
now 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 the
expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) to include children with cystic fibrosis (CF) ages 2
through 5 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 CF 6 years and older with at least one F508del
mutation or a mutation in the CFTR gene that is responsive to
TRIKAFTA® based on in vitro data.
“Since its initial approval, TRIKAFTA has had a significant
impact on the CF community, transforming the lives of thousands of
people living with cystic fibrosis,” said Carmen Bozic, M.D.,
Executive Vice President, Global Medicines Development and Medical
Affairs, and Chief Medical Officer, Vertex. “We remain steadfast in
our commitment to bringing highly effective CF treatments to people
of all ages living with this disease.”
This label expansion was supported by a 24-week Phase 3
open-label study which enrolled 75 children ages 2 through 5 years
old with CF to evaluate the safety, pharmacokinetics and efficacy
of TRIKAFTA®. The regimen was generally well tolerated, with a
safety profile consistent with that observed in older age groups,
and led to improvements in sweat chloride concentration, a measure
of CFTR function, and lung function. The data from this study were
recently published in the American Journal of Respiratory and
Critical Care Medicine.
“Early intervention with CFTR modulator therapies like TRIKAFTA
can offer the potential to improve the trajectory of CF lung
disease,” said Jennifer Goralski, M.D., Assistant Professor of
Medicine and Pediatrics, Co-Director, Adult Cystic Fibrosis Center,
University of North Carolina School of Medicine, and a lead
Principal Investigator in the TRIKAFTA® 2- to 5-year-old pivotal
clinical trial. “With this approval, we now have the ability to
treat young children with TRIKAFTA and can proactively address the
underlying cause of their disease.”
TRIKAFTA® was previously approved for the treatment of people
with CF ages 6 years and older with certain mutations in the U.S.,
Canada, Switzerland, Australia, New Zealand and Israel, as well as
in the EU, the U.K., Iceland, Liechtenstein and Norway as KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with
KALYDECO® (ivacaftor). Additionally, Vertex has submitted
applications for the use of TRIKAFTA®/KAFTRIO® in children ages 2
through 5 years of age to other global regulatory authorities,
including the European Medicines Agency (EMA) and the Medicines and
Healthcare Products Regulatory Agency (MHRA).
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 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.
INDICATIONS 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 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
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,
including, without limitation, statements made by Dr. Carmen Bozic
and Dr. Jennifer Goralski in this press release, statements
regarding the eligible patient population for TRIKAFTA®, including
newly eligible patient population, and statements regarding the
potential benefits 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 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 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 or Manisha Pai, +1 617-961-1899
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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