-Eligible patients in Northern Ireland 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 the Regional Pharmaceutical Procurement Service in
Northern Ireland has accepted an offer for access to 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 Northern Ireland ages 2 years and older who have two copies of
the F508del mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene will have access to 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 will have access to SYMKEVI® (tezacaftor/ivacaftor) in
combination with ivacaftor. We will support the arrangements being
put in place to ensure clinicians will be able to prescribe to
eligible patients within the next few weeks.
The agreement also offers expanded access to KALYDECO®
(ivacaftor) to include people ages 18 years and older who have the
R117H mutation and those patients ages 12 months and older who have
one of the nine licensed gating mutations.
“We are pleased that the nearly 280 eligible cystic fibrosis
patients in Northern Ireland will soon have access to CFTR
modulators to treat the underlying cause of their disease and we
thank the authorities in Northern Ireland for their collaboration
and commitment in this agreement,” said Ludovic Fenaux, Senior Vice
President, Vertex International.
About CF in the UK Over 10,000 people in the UK have CF —
the second highest number in the world. Nearly 480 people in
Northern Ireland 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 and statements regarding our
expectations for the patient populations that will be able to
access Vertex’s medicines and the timing of such access. 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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