European Commission Approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years
January 11 2022 - 3:01AM
Business Wire
- More than 1,500 children now eligible to
receive a treatment targeting the underlying cause of cystic
fibrosis for the first time -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted approval for the
label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a
combination regimen with ivacaftor for the treatment of cystic
fibrosis (CF) in patients ages 6 through 11 years old who have at
least one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
“We are delighted that KAFTRIO
(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with
ivacaftor is now approved for these young patients in the European
Union. It provides a new treatment option for physicians to help
treat the underlying cause of this devastating disease early in
life,” said Reshma Kewalramani, M.D., Chief Executive Officer and
President at Vertex. “This important milestone brings us one step
closer to our ultimate goal of developing treatments for all
patients living with CF.”
“Ivacaftor/tezacaftor/elexacaftor plus ivacaftor has shown
clinical benefit since its availability last year for people with
CF ages 12 and above,” said Professor Marcus A. Mall, M.D., Head of
the Department of Pediatric Respiratory Medicine, Immunology and
Critical Care Medicine at Charité University Medical Center Berlin.
“CF is a progressive disease, in which symptoms and organ damage
manifest very early in life. As a physician, I welcome the approval
of this medicine for this younger age group, as it will help us
treat eligible children with CF as early as 6 years old.”
As a result of long-term reimbursement agreements in Austria,
Northern Ireland and Denmark, and provisions for access in health
care systems such as Germany, eligible patients in these countries
will have access to the expanded indication for KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor) in a combination regimen with
ivacaftor shortly following regulatory approval by the European
Commission. Vertex will continue to work with reimbursement bodies
across the European Union to ensure access for all eligible
patients.
This medicine has also been approved by regulatory authorities
in New Zealand and in Switzerland, where it is known as TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor), for people with
CF ages 6 and above, and we continue to work closely with
reimbursement bodies in these countries to ensure access for all
eligible patients.
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 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 early 30s.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in A
Combination Regimen 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.
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 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 medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney disease. In addition, Vertex has a rapidly
expanding pipeline of cell and genetic therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
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, one of the 2021 Seramount (formerly Working Mother
Media) 100 Best Companies, and a best place to work for LGBTQ
equality by the Human Rights Campaign. For company updates and to
learn more about Vertex's history of innovation, visit
https://global.vrtx.com/ or follow us on Twitter and LinkedIn.
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 Reshma
Kewalramani, M.D., Chief Executive Officer and President at Vertex,
and Professor Marcus A. Mall, M.D., Head of the Department of
Pediatric Respiratory Medicine, Immunology and Critical Care
Medicine at Charité University Medical Center Berlin, in this press
release and statements regarding the estimated number of children
eligible for a medicine that can treat the underlying cause of
their disease for the first time, our beliefs regarding the
benefits of our medicines, and the anticipated patient access to
KAFTRIO® in combination with ivacaftor. 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 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
https://global.vrtx.com/ and www.sec.gov. 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)
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Media: mediainfo@vrtx.com or International: +44 20 3204
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