Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) as First & Only CFTR Modulator to Treat Eligible Inf...
November 05 2020 - 04:00AM
Business Wire
- Approval provides opportunity to treat the
underlying cause of cystic fibrosis earlier than ever before in
Europe -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted approval of the
label extension for KALYDECO® (ivacaftor) granules to include the
treatment of infants with cystic fibrosis (CF) ages 4 months and
older and weighing at least 5 kg who have the R117H mutation or one
of the following gating (class III) mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene: G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
“Our very first CFTR modulator, KALYDECO, was first approved
eight years ago, for certain CF patients ages 6 years and older.
With today’s approval, babies as young as 4 months are eligible and
we believe early treatment is important in managing CF,” said
Reshma Kewalramani, M.D., Chief Executive Officer and President,
Vertex. “Today’s approval is a testament to our commitment to keep
going until all people with CF have a treatment option.”
The label update is based on data from a cohort in the 24-week
Phase 3 open-label safety study (ARRIVAL) consisting of six
children with CF ages four months to less than six months who have
eligible gating mutations.
KALYDECO® (ivacaftor) will be now 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.
KALYDECO® (ivacaftor) is already approved in Europe for people
with CF ages 6 months and older weighing at least 5 kg who have one
of the following mutations in the CFTR gene: G551D, G1244E, G1349D,
G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.
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 KALYDECO® (ivacaftor)
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.
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 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, and statements regarding the
eligible patient population in Europe, our expectations regarding
the timing of access to KALYDECO for eligible patients four months
of age and older across countries in Europe, and our plans to
secure access to KALYDECO for additional eligible patients four
months of age and older 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 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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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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