-Application to add ~200 non-F508del CFTR
mutations to the KAFTRIO® license-
-If approved, ~2,800 people with cystic
fibrosis in the European Union ages 2 and above could receive a
medicine that treats the underlying cause of their disease for the
first time-
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the
European Medicines Agency (EMA) has validated a Type II variation
application to the Marketing Authorization for KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor.
The application is for expansion of the approved indication for
KAFTRIO® in a combination regimen with ivacaftor for the treatment
of people with cystic fibrosis (CF) ages 2 and above who have a
mutation in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene that is responsive based on clinical and/or in vitro
data, including the N1303K mutation. The application will now be
reviewed by the Committee for Medicinal Products for Human Use
(CHMP), which will issue an opinion to the European Commission
regarding the potential approval of this license expansion.
Data to support this submission includes the results of a Phase
3, randomized, placebo-controlled clinical study in people with
rare non-F508del KAFTRIO®-responsive CFTR mutations. This study met
its primary endpoint and showed that KAFTRIO® in combination with
ivacaftor resulted in rapid, statistically significant, and
clinically meaningful improvements in lung function compared to
placebo (9.2 percentage point increase in ppFEV1; P<0.0001; 95%
CI [7.2, 11.3]). The medicine was generally well tolerated, with
safety data generally consistent with the established safety
profile of KAFTRIO® in combination with ivacaftor.
The Marketing Authorization Application submission package also
includes real-world evidence data from the U.S. Cystic Fibrosis
Foundation Patient Registry with respect to people with CF with
non-F508del KAFTRIO®-responsive CFTR mutations who are receiving
commercially available TRIKAFTA® (which is the name for KAFTRIO® in
the U.S.). In addition, the submission includes in vitro data using
a well-established laboratory model that has been the basis of
approval of the rare mutations indication in the U.S.
“It is encouraging to see such positive clinical trial results
for KAFTRIO in people with CF with these rare types of mutations,
which are non-F508del,” said Professor Isabelle Fajac, Professor of
Physiology, Cochin Hospital, Assistance Publique-Hôpitaux de Paris,
Université Paris Cité, Paris, France. “The majority of these people
currently have no treatment option to address the underlying cause
of their CF, so this submission is an extremely important step
towards a medicine becoming available for these people with high
unmet medical needs.”
“We are committed to going the distance in cystic fibrosis and
dedicated to bringing treatments to all people with CF,” said Nia
Tatsis, Ph.D., Executive Vice President, Chief Regulatory and
Quality Officer at Vertex. “We look forward to working with the EMA
on this important submission for people with CF who have
non-F508del KAFTRIO-responsive rare mutations, who currently cannot
access KAFTRIO for the underlying cause of their disease.”
Vertex plans to submit regulatory filings for the same mutations
in Australia, Brazil, Canada, New Zealand and Switzerland. The
company also plans to submit a subset of these mutations, including
N1303K and non-canonical splice mutations, not currently included
in the U.S. TRIKAFTA® label to the U.S. FDA.
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 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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in
Combination With 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. KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor) in combination with 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 ivacaftor,
tezacaftor and elexacaftor help hydrate and clear mucus from the
airways.
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with
ivacaftor is approved in the European Union for the treatment of
cystic fibrosis (CF) in patients aged 2 years and older who have at
least one copy of the F508del mutation in the CFTR gene.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
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. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, YouTube and Twitter/X.
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 Nia Tatsis,
Ph.D., and Professor Isabelle Fajac in this press release and
statements regarding our expectation that, if approved for these
mutations, 2,800 people with CF in the EU ages 2 and above could
receive a medicine that treats the underlying cause of their
disease for the first time, our expectations for a CHMP opinion
regarding potential approval of this license expansion for KAFTRIO,
statements regarding the potential benefits of KAFTRIO, our plans
to submit regulatory filings for the same mutations in Australia,
Brazil, Canada, New Zealand and Switzerland, and our plans to
submit regulatory filings for a subset of these mutations to the
U.S. FDA for the TRIKAFTA label. 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 factors that could cause actual events or results to
differ materially from those indicated by such forward-looking
statements. Those risks and uncertainties include, among other
things, that data from the company’s development programs may not
support a label expansion for KAFTRIO/TRIKAFTA, that regulatory
authorities may not approve a label expansion for KAFTRIO/TRIKAFTA
on a timely basis or at all, and other risks listed under the
heading “Risk Factors” in Vertex's annual report and in subsequent
filings filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com and
www.sec.gov. 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)
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