- The combination therapy is a new treatment
option for CF patients who are homozygous for F508del -
- The only medicine to treat the underlying
cause of CF in this age group with one F508del mutation and one of
14 residual function mutations -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted approval of the
label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO®
(ivacaftor), to include the treatment of cystic fibrosis (CF) in
patients ages 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 one
of 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.
“With this approval, children with CF in Europe ages 6 to 11
years with the most common mutation, F508del, have a new treatment
option and children with certain residual function mutations will,
for the first time, have a treatment option available that
addresses the underlying cause of their CF,” said Reshma
Kewalramani, M.D., Chief Executive Officer and President at Vertex.
“Today’s approval brings us closer to our ultimate goal of
providing medicines for all people with CF.”
SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) will
be immediately available to additional eligible patients in Germany
and will be available shortly in countries that have entered into
innovative long-term reimbursement agreements with Vertex,
including the UK, Denmark and the Republic of Ireland. In all other
countries, Vertex will work closely with relevant authorities in
Europe to secure access for eligible patients.
In Europe, SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO®
(ivacaftor) is already approved for the treatment of people with CF
ages 12 years and older who have either two copies of the F508del
mutation in the CFTR gene, or one copy of the F508del mutation and
a copy of one of the following 14 mutations in which the CFTR gene
results in residual CFTR protein activity: P67L, R117C, L206W,
R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H,
2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease
affecting approximately 75,000 people worldwide. CF is a
progressive, multi-system disease that affects the lungs, liver, GI
tract, sinuses, sweat glands, pancreas 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. While
there are many different types of CFTR mutations that can cause the
disease, the vast majority of all people with CF have at least one
F508del mutation. These mutations, which can be determined by a
genetic test, or genotyping test, lead to CF by creating
non-working and/or too few CFTR proteins 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 that can cause chronic lung infections and
progressive lung damage in many patients that eventually leads to
death. The median age of death is in the early 30s.
About SYMKEVI® in combination with KALYDECO®
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.
For complete product information including dosing guidance,
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 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 medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney diseases. In addition, Vertex has a rapidly
expanding pipeline of genetic and cell therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
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 11 consecutive years on Science magazine's Top
Employers list and a best place to work for LGBTQ equality by the
Human Rights Campaign. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
Facebook, Twitter, LinkedIn, YouTube and Instagram.
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 Dr. Reshma
Kewalramani in this press release, statements regarding the
eligible patient population in Europe, our expectations regarding
the timing of access to SYMKEVI in combination with KALYDECO for
eligible patients ages 6-11 years across countries in Europe, and
our plans to secure access to SYMKEVI in combination with KALYDECO
for additional eligible patients ages 6-11 years in Europe. 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, risks related to commercializing SYMKEVI
in combination with KALYDECO in Europe, and other risks listed
under Risk Factors in Vertex's most recent annual report and
subsequent quarterly reports filed with the Securities and Exchange
Commission and available through the company's website at
www.vrtx.com. 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:
InvestorInfo@vrtx.com or 617-961-7163
Media: mediainfo@vrtx.com or International: +44 20 3204
5275 or U.S.: 617-341-6992
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