TORONTO, Sept. 26,
2024 /CNW/ - Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that Health Canada
has accepted for review its New Drug Submission (NDS) for
vanzacaftor/tezacaftor/deutivacaftor, a once-daily triple
combination therapy for people living with cystic fibrosis (CF)
ages 6 years and older who have at least one F508del
mutation or another responsive mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene.
"Vanzacaftor raises the bar set by TRIKAFTA® by
demonstrating comparable lung function improvement and additional
improvements in CFTR function, as measured by sweat chloride in
Phase 3 trials," said Michael Siauw, General Manager at Vertex
Pharmaceuticals (Canada)
Incorporated. "Health Canada's
acceptance of the vanzacaftor submission for review brings us one
step closer to providing a new treatment option, with potential for
additional benefit, for eligible people living with CF."
The NDS will be part of an aligned review with Canadian Health
Technology Assessment (HTA) organizations, Canada's Drug Agency (CDA) and the Institut
national d'excellence en santé et en services sociaux (INESSS)
in Quebec.
Vanzacaftor is also undergoing regulatory review in multiple
other jurisdictions, including by the Food and Drug Administration
(FDA) in the U.S. and by the European Medicines Agency (EMA) in the
E.U.
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 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.
Diagnosis of CF is often made by genetic testing and is
confirmed by testing sweat chloride (SwCl), which measures CFTR
protein dysfunction. The diagnostic threshold for CF is SwCl ≥60
mmol/L, while levels between 30-59 indicate CF is possible and more
testing may be needed to make the diagnosis of CF. A SwCl level of
<30 mmol/L is seen in people who carry one copy of a CFTR gene
mutation but do not have any manifestation of disease (carriers).
Higher levels of SwCl are associated with more severe disease.
Restoring CFTR function leads to lower levels of SwCl. Restoring
SwCl levels below 30 mmol/L has long been the ultimate treatment
goal for Vertex, as levels below 30 mmol/L are considered normal
and are typical of CF carriers who do not have disease.
About vanzacaftor/tezacaftor/deutivacaftor (the "vanza
triple")
In people with CF, mutations in the CFTR gene lead to decreased
quantity and/or function of the CFTR protein channel at the cell
surface. Vanzacaftor and tezacaftor are designed to increase the
amount of CFTR protein at the cell surface by facilitating the
processing and trafficking of the CFTR protein. Deutivacaftor is a
potentiator designed to increase the channel open probability of
the CFTR protein delivered to the cell surface to improve the flow
of salt and water across the cell membrane. The vanza triple
remains investigational in Canada,
and the safety and efficacy have not been established by Health
Canada.
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 and Latin America. 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.
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 by Michael Siauw in this press release, and
statements regarding our expectations for the benefits of and
potential for vanzacaftor and our expectations for the NDS. 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)
Vertex Pharmaceuticals Incorporated
Investors:
Susie Lisa, +1 617-341-6108
or
Manisha Pai, +1 617-961-1899
or
Miroslava Minkova, +1
617-341-6135
Media:
mediainfo@vrtx.com
or
Canada: +1 647-790-1600
SOURCE Vertex Pharmaceuticals (Canada) Inc.
