- With this reimbursement agreement in place,
more than 2,200 people with CF will have PBS-funded access to
TRIKAFTA®, including more than 700 who will now have access to a
CFTR modulator therapy for the first time -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that as of 1 April 2022, TRIKAFTA®
(elexacaftor/tezacaftor/ivacaftor and ivacaftor) will be reimbursed
on the Australian Pharmaceutical Benefits Scheme (PBS) for the
treatment of cystic fibrosis (CF) in people ages 12 years and older
who have at least one F508del mutation in the CFTR gene, the most
common CF-causing mutation worldwide.
“Today’s announcement is a significant milestone in ensuring
Australians living with CF receive timely and sustainable access to
TRIKAFTA,” said Ludovic Fenaux, Senior Vice President, Vertex
International. “This is the fourth treatment we have brought to
Australians over the last eight years, working tirelessly alongside
the CF patient and clinical communities. We thank the Australian
Government for recognizing the significant need for TRIKAFTA and
the value it brings.”
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was
approved by the Australian Therapeutic Goods Administration (TGA)
in March 2021 based on the results of four global Phase 3 clinical
trials, which included multiple Australian trial sites and
patients.
CF is a rare, life-shortening, genetic disease affecting
approximately 3,500 people in Australia. It is caused by a
defective and/or missing CFTR protein, resulting from mutations in
the CFTR gene. Up to 90 percent of people living with CF have at
least one F508del mutation.
“As a genetic disease, cystic fibrosis is a prime candidate for
precision medicine. Now, with PBS listing of TRIKAFTA, eligible
Australians living with CF ages 12 years and older can broadly
access a therapy that treats the underlying cause of their disease.
Clinicians across Australia will be excited about this most welcome
news,” said Professor John Wilson AM, Head, Cystic Fibrosis
Service, Alfred Health Australia.
Australia now joins the list of 30 countries where the triple
combination therapy is approved and reimbursed including Denmark,
Finland, France, Germany, Italy, Ireland, Israel, Poland, Spain,
Switzerland and the countries within the U.K.
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 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. TRIKAFTA®
(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.
For complete product information, please see the Summary of
Product Characteristics that can be found on
https://www.tga.gov.au/apm-summary/trikafta.
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
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 Ludovic Fenaux,
Senior Vice President, Vertex International, and Professor John
Wilson AM, Head, Cystic Fibrosis Service, Alfred Health, in this
press release and statements regarding the reimbursement of and
access to TRIKAFTA® for certain patients, the estimated number of
patients eligible for a CFTR modulator therapy in Australia,
including patients that will now have access to a CFTR modulator
therapy for the first time, and our beliefs about the benefits of
our medicines. 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 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
registration or further development of its compounds due to safety,
efficacy, or other reasons, risks related to obtaining approval for
and commercializing our medicines, and other risks listed under the
heading “Risk Factors” in Vertex's annual report filed with the
Securities and Exchange Commission (SEC) and available through the
company's website at https://global.vrtx.com/ and on the SEC’s
website at 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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Vertex Pharmaceuticals Incorporated
Investors: Michael Partridge, +1 617-341-6108 or Manisha
Pai, +1 617-429-6891
Media: mediainfo@vrtx.com or International: +44 20 3204
5275 or U.S.: +1 617-341-6992
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