-Approximately 1,100 F/MF patients now
eligible for a CFTR modulator to treat the underlying cause of
their disease-
TORONTO, June 18, 2021 /CNW/ - Vertex Pharmaceuticals
(Canada)
Incorporated (NASDAQ: VRTX) today announced Health Canada has
granted Marketing Authorization for PrTRIKAFTA®
(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)
Vertex Pharmaceuticals Incorporated
SOURCE Vertex Pharmaceuticals (Canada) Inc.