Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor & ivacaftor) in People Ages 12 ...
June 18 2021 - 3:37PM
Business Wire
-Approximately 1,100 F/MF patients now eligible
for a CFTR modulator to treat the underlying cause of their
disease-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced Health Canada has granted Marketing Authorization for
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the
treatment of cystic fibrosis (CF) in people ages 12 years and older
who have at least one F508del mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, the most common
CF-causing mutation. With this approval, for the first time,
approximately 1,100 eligible patients with CF ages 12 years and
older who have at least one F508del mutation have a medicine that
targets the underlying cause of their CF.
“The approval of TRIKAFTA marks a significant milestone for
Canadians with CF, their families and Vertex,” said Reshma
Kewalramani, M.D., Chief Executive Officer and President, Vertex.
“I would like to thank the people with CF who participated in our
clinical trials, our dedicated scientists and the investigators who
have enabled this innovative medicine to be approved in Canada
today. Without their commitment, this milestone would not have been
possible.”
“I have seen substantial improvements in patients treated with
TRIKAFTA in clinical practice, including improved lung function,”
said Dr. Elizabeth Tullis, Medical Director, Toronto Adult CF
Centre, Professor of Medicine, University of Toronto. “I’m excited
that more Canadians may be able to benefit from CFTR modulators and
look forward to seeing the impact of this medicine for all patients
who can benefit from it.”
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. 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 three global Phase 3 studies in people ages 12
years and older with CF: a 24-week Phase 3 study (Study 445-102) in
403 people with one F508del mutation and one minimal function
mutation (F/MF), a four-week Phase 3 study (Study 445-103) in 107
people with two F508del mutations (F/F), and a Phase 3 study (Study
445-104) in 258 people heterozygous for the F508del-CFTR mutation
and a CFTR gating mutation (F/G) or a residual function mutation
(F/RF).
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.
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.
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. Kewalramani
and Dr. Tullis in this press release, and statements regarding our
expectations for eligible patient population and access to
TRIKAFTA® and our beliefs regarding the benefits of our medicine.
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 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. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
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mediainfo@vrtx.com or Colin Le Fevre: +1 416-320-0316
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