- If accepted by the Scottish Medicines
Consortium (SMC) for use on the NHS in Scotland, eligible patients
could have access to these precision cystic fibrosis medicines in
2019 -
- While the SMC reviews the submissions,
clinicians can apply for access to these CF medicines via the PACS
Tier 2 process for individual patients based on clinical need -
- Vertex and the Scottish Government have
agreed a confidential discount which would be applied to approved
PACS Tier 2 applications -
Vertex Pharmaceuticals (Europe) Limited today announced that,
following constructive discussions with the Scottish Government, it
will submit ORKAMBI® (lumacaftor/ivacaftor) as well as SYMKEVI®
(tezacaftor/ivacaftor) to be used in combination with ivacaftor, to
the Scottish Medicines Consortium (SMC) for appraisal. If accepted
by the SMC for use on the NHS in Scotland, eligible patients with
cystic fibrosis (CF) in Scotland could have access to these
precision medicines in 2019.
“Our recent conversations with the Scottish Government, and the
SMC’s orphan medicines process have provided important flexibility
for evaluating precision medicines, such as cystic fibrosis
transmembrane conductance regulator (CFTR) modulators. Their
methods reflect the innovative nature of medicines that have the
potential to extend life for patients with rare diseases, like CF,”
said Ludovic Fenaux, Senior Vice President, Vertex International.
“We are hopeful that, through this process, all eligible patients
in Scotland could have access to our medicines soon.”
While the SMC reviews the submissions, clinicians could apply
for access to lumacaftor/ivacaftor as well as tezacaftor/ivacaftor
in combination with ivacaftor for individual patients based on
clinical need via the Peer Approved Clinical System (PACS) Tier 2
process for ‘individual access to medicines not yet generally
available on the NHS’. Vertex and the Scottish Government have also
agreed to a confidential discount which will be applied to
applications to the PACS Tier 2 process.
Approximately 900 people in Scotland live with CF.1 In the UK,
the median age of death is 31 years.2 NHS Scotland estimates that
one in 24 Scots have a CFTR mutation which, if carried by both
parents, would lead to a child being born with CF.3
Should the appraisal be positive, patients in Scotland will join
those in other countries around the world where all those eligible
have access to lumacaftor/ivacaftor, including Austria, Australia,
Denmark, Germany, the Republic of Ireland, Italy, the Netherlands,
Sweden and the U.S.
About cystic fibrosisCystic fibrosis (CF) is a rare,
life-shortening genetic disease affecting approximately 75,000
people in North America, Europe and Australia.4
CF is caused by a defective or missing cystic fibrosis
transmembrane conductance regulator (CFTR) protein resulting from
mutations in the CFTR gene. Children must inherit two defective
CFTR genes — one from each parent — to have CF. There are
approximately 2,000 known mutations in the CFTR gene.5 Some of
these mutations, which can be determined by a genetic test, or
genotyping test, lead to CF by creating non-working or too few CFTR
proteins at the cell surface. The defective function or absence of
CFTR protein results in poor flow of salt and water into and out of
the cell in a number of organs. In the lungs, this leads to the
build-up of abnormally thick, sticky mucus that can cause chronic
lung infections and progressive lung damage in many patients that
eventually leads to death.6
About ORKAMBI® (lumacaftor/ivacaftor) and the
F508del mutationIn 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. Patients with two copies of the F508del mutation are
easily identified by a simple genetic test.
Lumacaftor/ivacaftor is a combination of lumacaftor, which 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, and ivacaftor, which is designed to enhance
the function of the CFTR protein once it reaches the cell surface.
Lumacaftor/ivacaftor is typically taken twice per day.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About SYMKEVI® (tezacaftor/ivacaftor) in
combination with ivacaftorSome mutations result in CFTR protein
that is not processed or folded normally within the cell, and that
generally does not reach the cell surface. Tezacaftor is designed
to address the trafficking and processing defect of the CFTR
protein to enable it to reach the cell surface and ivacaftor is
designed to enhance the function of the CFTR protein once it
reaches the cell surface.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About VertexVertex is a global biotechnology company that
invests in scientific innovation to create transformative medicines
for people with serious and life-threatening diseases. In addition
to clinical development programs in CF, Vertex has more than a
dozen ongoing research programs focused on the underlying
mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including being named to Science magazine's Top Employers in
the life sciences ranking for nine years in a row. For additional
information and the latest updates from the company, please visit
www.vrtx.com.
Special Note Regarding Forward-looking StatementsThis
press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including,
without limitation, the statements in the first through third
paragraphs of this press release. While Vertex believes the
forward-looking statements contained in this press release are
accurate, 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, risks related to commercializing our products
and the other risks listed under Risk Factors in Vertex's annual
report and quarterly reports filed with the Securities and Exchange
Commission. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
1 CF Registry Scotland Report 2015 Available at:
https://www.cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/reporting-and-resources
(Accessed: December 2018)2 UK Cystic Fibrosis Registry Annual Data
Report 2016 Available at:
https://www.cysticfibrosis.org.uk/~/media/documents/the-work-we-do/uk-cf-registry/uk-cf-registry-annual-data-report-2016.ashx?la=en
(Accessed: December 2019)3 National Services Scotland. Adult Cystic
Fibrosis. Available at:
http://www.nsd.scot.nhs.uk/services/specserv/adultcf.html
(Accessed: December 2018)4 Basharut A. S. et al. The cystic
fibrosis drug market, Nature Reviews Drug Discovery vol.13, p.
721–722.2014 https://doi.org/10.1038/nrd44345 CF Trust. What are
the causes of CF. Available at:
https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/what-causes-cystic-fibrosis
(Accessed: December 2018)6 Real-world outcomes in patients with
cystic fibrosis treated with ivacaftor: 2016 US and UK cystic
fibrosis Registry analyses.” Poster IPD2.02 during Session
IPD2--What do We Learn from CFTR Modulator Use in Real Life. Poster
IPD2.01 during Session IPD2. Poster presented at 41st European
Cystic Fibrosis Conference, June 6-9, 2018, in Belgrade, Serbia
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Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1-617-341-6108orEric Rojas, +1-617-961-7205orZach Barber,
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