- Agreement includes access to any future
licence extensions -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced an extended long-term reimbursement agreement with NHS
England providing access to KAFTRIO®
(ivacaftor/tezacaftor/elexacaftor), SYMKEVI® (tezacaftor/ivacaftor)
and ORKAMBI® (lumacaftor/ivacaftor) for all existing and future
eligible cystic fibrosis (CF) patients in England.
The agreement, which also includes access to any future licence
extensions of these medicines, comes as the National Institute for
Health and Care Excellence (NICE) issues a positive recommendation
for these CFTR modulators.
Vertex, NHS England and NICE have also committed to work
together on a path towards rapid access for all eligible patients
for the next in class triple combination treatment for CF, the
investigational therapy vanzacaftor/tezacaftor/deutivacaftor,
subject to authorisation by the Medicines and Healthcare products
Regulatory Agency (MHRA).
Vertex is working with the NHS authorities in Scotland, Wales
and Northern Ireland to finalise similar access agreements as soon
as possible.
“We are delighted to have agreed extended long-term access to
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), SYMKEVI®
(tezacaftor/ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) for
eligible CF patients in England,” said Ludovic Fenaux, Senior Vice
President, Vertex International. ”I would like to acknowledge the
collaboration of NHS England, NICE and the SMC, and also thank the
CF community for their contribution in describing the value that
these innovative medicines bring to patients.”
Vertex CF medicines are broadly available in over 60 countries
worldwide including Australia, France, Italy, Germany, the Republic
of Ireland, the Netherlands, Spain and the U.S.
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 a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 2 years and older who have at
least one F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
For complete product information, please see the Summary of
Product Characteristics that can be found on
https://products.mhra.gov.uk/.
About SYMKEVI® (tezacaftor/ivacaftor) 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. SYMKEVI® (tezacaftor/ivacaftor) in combination with
ivacaftor is an oral medicine that is a combination of tezacaftor
and ivacaftor. Tezacaftor is designed to increase the amount of
mature protein at the cell surface by targeting the trafficking and
processing defect of the CFTR protein to enable it to reach the
cell surface. 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
tezacaftor and ivacaftor help hydrate and clear mucus from the
airways.
SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with
KALYDECO® (ivacaftor) is a prescription medicine used for the
treatment of cystic fibrosis (CF) in patients aged 6 years and
older who have two copies of the F508del mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene or one
copy of the F508del mutation and one copy of the following 14
mutations in the CFTR gene that result in residual CFTR activity:
P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F,
R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T.
For complete product information, please see the Summary of
Product Characteristics that can be found on
https://products.mhra.gov.uk/.
About ORKAMBI® (lumacaftor/ivacaftor)
In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little to no CFTR protein at the cell surface.
ORKAMBI® (lumacaftor/ivacaftor) is an oral medicine that is a
combination of lumacaftor and ivacaftor. Lumacaftor is designed to
increase the amount of mature protein at the cell surface by
targeting the processing and trafficking defect of the F508del-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
lumacaftor and ivacaftor help hydrate and clear mucus from the
airways.
ORKAMBI® (lumacaftor/ivacaftor) is a prescription medicine used
for the treatment of cystic fibrosis (CF) in patients aged 1 year
and older who have two copies of the F508del mutation
(F508del/F508del) in their CFTR gene.
For complete product information, please see the Summary of
Product Characteristics that can be found on
https://products.mhra.gov.uk/.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 92,000 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.
Diagnosis of CF is often made by genetic testing and is
confirmed by testing sweat chloride (SwCl), which measures CFTR
protein dysfunction. The diagnostic threshold for CF is SwCl ≥60
mmol/L, while levels between 30-59 indicate CF is possible and more
testing may be needed to make the diagnosis of CF. A SwCl level of
<30 mmol/L is seen in people who carry one copy of the CF gene
but do not have any manifestation of disease (carriers). Higher
levels of SwCl are associated with more severe disease. Restoring
CFTR function leads to lower levels of SwCl.
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 acute and neuropathic
pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal
dominant polycystic kidney disease, type 1 diabetes, myotonic
dystrophy type 1 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, Latin America and the
Middle East. 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/en-gb/
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Vertex Pharmaceuticals (NASDAQ:VRTX)
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