-Eligible patients in Wales will soon have
access to ORKAMBI® (lumacaftor/ivacaftor) and
SYMKEVI® (tezacaftor/ivacaftor), expanded access to
KALYDECO® (ivacaftor) under same terms as NHS England
agreement-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
confirms that NHS Wales has accepted an offer for all currently
licensed Vertex cystic fibrosis (CF) medicines and any future
indications of these medicines under the same terms as the recently
announced agreement with NHS England.
This means that once the contract is finalized, patients with CF
in Wales ages 2 years and older who have two copies of the F508del
mutation in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene can access ORKAMBI® (lumacaftor/ivacaftor) and CF
patients ages 12 years and older who either have two copies of the
F508del mutation or one copy of the F508del mutation and a copy of
one of the other 14 licensed mutations can access SYMKEVI®
(tezacaftor/ivacaftor) in combination with ivacaftor in the coming
weeks.
The agreement also offers expanded access to KALYDECO®
(ivacaftor) to include those patients ages 12 months and older who
have one of the nine licensed gating mutations.
“Today’s announcement is good news for the approximately 270
eligible cystic fibrosis patients in Wales who will soon have
access to CFTR modulators to treat the underlying cause of their
disease,” said Ludovic Fenaux, Senior Vice President, Vertex
International. “We thank the authorities in Wales for their
collaboration in accepting this offer under the same terms as were
recently announced in England.”
About CF in the UK Over 10,000 people in the UK have CF —
the second highest number in the world. Over 430 people in Wales
have CF. CF is a debilitating, life-shortening inherited condition
that causes progressive damage to organs across the body from
birth. Currently, there is no cure for CF and half of people in the
UK with CF die before they are 32. The daily impact of treatment is
significant. It can take up to four or more hours, involving
nebulizers, physiotherapy and up to 70 tablets a day. CF accounts
for 9,500 hospital admissions and over 100,000 hospital bed days a
year. A third of these are used by children under 15.
About ORKAMBI® (lumacaftor/ivacaftor) and the F508del
mutation 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.
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.
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
ivacaftor Some 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.
SYMKEVI is indicated for people with CF ages 12 and older who
either have two copies of the F508del mutation or one copy of the
F508del mutation and have one of the following 14 mutations in
which the CFTR protein shows residual function: P67L, R117C, L206W,
R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H,
2789+5G→A, 3272-26A→G, or 3849+10kbC→T.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu.
About KALYDECO® (ivacaftor) KALYDECO® (ivacaftor) is the
first medicine to treat the underlying cause of CF in people with
specific mutations in the CFTR gene. Known as a CFTR potentiator,
ivacaftor is an oral medicine designed to keep CFTR proteins at the
cell surface open longer to improve the transport of salt and water
across the cell membrane, which helps hydrate and clear mucus from
the airways.
KALYDECO is indicated in people ages 12 months and older who
have one of the following mutations in the CFTR gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
KALYDECO is also indicated for the treatment of patients with CF
ages 18 years and older who have an R117H 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 four 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 medicines in other serious
diseases where it has deep insight into causal human biology, such
as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin
deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney
diseases.
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, UK. 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 10 consecutive years on Science magazine's Top
Employers list and top five on the 2019 Best Employers for
Diversity list by Forbes.
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, the statements by Mr. Fenaux in the fourth
paragraph of this press release, statements regarding our
expectations for the patient populations that will be able to
access Vertex’s medicines and the timing of such access, and
statements about our expectations regarding a formal agreement in
Northern Ireland. 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 expressed or implied 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, and other risks
listed under Risk Factors in Vertex's annual report and subsequent
quarterly reports filed with the Securities and Exchange Commission
and available through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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