-Approximately 200 people with certain rare CF
mutations are now eligible for TRIKAFTA®-
TORONTO, July 15,
2024 /CNW/ - Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that Health Canada
has granted Marketing Authorization for the expanded use of
PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor
and ivacaftor) for the treatment of cystic fibrosis (CF) in
patients aged 2 years and older who have a mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene
that is responsive based on clinical and/or in vitro data.
TRIKAFTA® was previously approved by Health Canada for
use in people with CF aged 2 years and older with at least one
F508del mutation and is now approved for 152 additional
mutations.
"This expanded approval of TRIKAFTA for patients with a
responsive mutation underscores the commitment of our scientists to
the development of medicines for all people living with CF," said
Michael Siauw, General Manager at
Vertex Pharmaceuticals (Canada)
Incorporated. "We remain dedicated to the CF community and are
excited for the hope this approval brings to newly eligible CF
patients and families across the country."
"The approval of TRIKAFTA for certain non-F508del
mutations is a transformative moment for CF management in
Canada," said Elizabeth Tullis, M.D., Director of the Toronto
Adult CF Clinic at St. Michael's
Hospital, Unity Health Toronto and Professor of Medicine,
University of Toronto. "Many patients
not previously eligible for CFTR modulators may now be able to
benefit from a treatment that targets the underlying cause of their
disease for the first time."
The label update is based on data from multiple sources,
including a 24-week randomized placebo-controlled double-blind
study in patients aged 6 years and older who had at least one
qualifying non-F508del mutation to evaluate the
safety and efficacy of TRIKAFTA®. The study met its
primary endpoint, TRIKAFTA® was generally
well tolerated, and safety data were similar to those
observed in previous studies. Additionally, published clinical
data and robust in vitro data supported the expanded
indication.
The full list of indicated mutations for TRIKAFTA can be found
in the Health Canada-approved Product Monograph.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 92,000 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 the 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 30s, but with treatment, projected survival
is improving.
Today Vertex CF medicines are treating over 65,000 people with
CF across 60 countries on six continents. This represents 2/3 of
the diagnosed people with CF eligible for CFTR modulator
therapy.
About PrTRIKAFTA®
(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.
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) 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, or another mutation
responsive to TRIKAFTA®, in the 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 2 years of age.
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 approved medicines that
treat the underlying causes of multiple chronic, life-shortening
genetic diseases — cystic fibrosis, sickle cell disease and
transfusion-dependent beta thalassemia — and continues to advance
clinical and research programs in these diseases. Vertex also has a
robust clinical pipeline of investigational therapies across a
range of modalities in other serious diseases where it has deep
insight into causal human biology, including acute and neuropathic
pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal
dominant polycystic kidney disease, type 1 diabetes, myotonic
dystrophy type 1 and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international
headquarters in London.
Additionally, the company has research and development sites and
commercial offices in North
America, Europe,
Australia, Latin America and the Middle East. Vertex is consistently recognized
as one of the industry's top places to work, including 14
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.ca or follow us on LinkedIn, YouTube and
Twitter/X.
(VRTX-GEN)
Special Note Regarding Forward-Looking
Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, statements made by
Michael Siauw and Elizabeth Tullis M.D. in this press release, and
statements regarding the number of people with certain rare
CF mutations expected to be eligible for TRIKAFTA in Canada. 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 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)
SOURCE Vertex Pharmaceuticals (Canada) Inc.