TORONTO, Nov. 29,
2023 /CNW/ - Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that Health Canada
has granted Market Authorization for the expanded use of
PrKALYDECO® (ivacaftor) for the
treatment of cystic fibrosis (CF) in children ages 2 months
and older weighing at least 3 kg who have one of the following
mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene: G551D, G1244E,
G1349D, G178R, G551S, S1251N,
S1255P, S549N, S549R or R117H.
"For the first time, children with specific mutations will be
eligible at 2 months of age for a medicine that treats the
underlying cause of their cystic fibrosis," said Michael Siauw, General Manager at Vertex
Pharmaceuticals (Canada)
Incorporated. "We remain committed to researching and developing
medicines for younger CF patients, and this important approval
highlights our continued dedication and progress."
Vertex will work closely with government and private payers
to facilitate access for eligible patients as soon as possible.
PrKALYDECO® was previously approved by
Health Canada for use in people with CF ages 4 months and older who
have one of the following mutations in
the CFTR gene: G551D, G1244E, G1349D,
G178R, G551S, S1251N, S1255P,
S549N, S549R or R117H.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 88,000 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.
About
PrKALYDECO® (ivacaftor)
In people with certain types of mutations in the CFTR
gene, the CFTR protein at the cell surface does not function
properly. Known as a CFTR potentiator, ivacaftor is an oral
medicine designed to facilitate the ability of CFTR proteins to
transport salt and water across the cell membrane, which helps
hydrate and clear mucus from the airways.
PrKALYDECO® (ivacaftor) was the first
medicine to treat the underlying cause of CF in people with
specific mutations in the 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 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 APOL1-mediated kidney
disease, acute and neuropathic pain, type 1 diabetes 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.
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 in this press release,
and statements regarding our plans to work closely with government
and private payers to facilitate access for eligible patients as
soon as possible. 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.