TORONTO, July 7, 2022
/CNW/ - Vertex Pharmaceuticals Incorporated (Canada) (Nasdaq: VRTX) today announced
that it has signed a Letter of Intent (LOI) with the pan-Canadian
Pharmaceutical Alliance (pCPA), which represents an agreement in
principle regarding the public reimbursement of
PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and
ivacaftor) for patients ages 6 years and older who have at least
one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
This is an amendment of the previous LOI which includes
TRIKAFTA® for ages 12 years and older,
PrKALYDECO® (ivacaftor), and
PrORKAMBI® (lumacaftor/ivacaftor). This LOI
amendment follows the positive clinical recommendation of
TRIKAFTA® for people with cystic fibrosis (CF) ages 6
years and older by the Canadian Agency for Drugs and Technology in
Health (CADTH). CADTH's recommendation has removed the 90%
ppFEV1 (percent predicted forced expiratory volume in 1
second) initiation criteria for all eligible patients 6 years and
older. This new recommendation supersedes CADTH's prior
recommendation of TRIKAFTA® for those ages 12 years and
older.
"This is a significant milestone for all people living with
cystic fibrosis in Canada,"
said Michael Siauw, General Manager,
Vertex Pharmaceuticals (Canada).
"We would like to thank CADTH, the pCPA and the participating
jurisdictions for their continued collaboration. CADTH's positive
clinical recommendation is consistent with the best practices of CF
management and extends to all eligible patients 6 years and
older. We will work quickly with the public drug plans to
apply this positive clinical recommendation so that all eligible
people living with CF have the opportunity to receive
TRIKAFTA."
The Institut national d'excellence en santé et en services
sociaux (INESSS) in Quebec is
currently conducting its health technology assessment of
TRIKAFTA® for patients ages 6 years and older. It is
anticipated that this recommendation will be published later this
year.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 83,000 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.
PrTRIKAFTA® (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 by binding to different sites on
the CFTR protein. 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® is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients ages 6 years and
older who have at least one copy of the F508del mutation in
the cystic fibrosis transmembrane conductance regulator
(CFTR) gene.
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, cell and genetic
therapies in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes,
alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
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 12 consecutive years on
Science magazine's Top Employers list and one of the 2021 Seramount
(formerly Working Mother Media) 100 Best Companies. 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 Michael Siauw in this press release, statements
regarding our expectations that eligible people with CF in
Canada will have access to
TRIKAFTA®, and statements regarding additional
recommendations expected to be made by Canadian authorities. 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 the
company ultimately may not be able to secure reimbursement in
Canada, 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)
SOURCE Vertex Pharmaceuticals (Canada) Inc.