Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old
April 27 2023 - 02:00AM
Business Wire
- If approved by the European Commission,
nearly 300 children with cystic fibrosis and two copies of the
F508del mutation would be eligible for the first time for a
medicine that can treat the underlying cause of their disease -
Vertex Pharmaceuticals (Nasdaq: VRTX) today
announced that the European Medicines Agency’s (EMA) Committee for
Medicinal Products for Human Use (CHMP) adopted a positive opinion
for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the
treatment of children with cystic fibrosis (CF) ages 1 to <2
years old who have two copies of the F508del mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene, the most
common form of the disease.
“Symptoms and organ damage start very early in the lives of
people with cystic fibrosis. Treating patients as young as possible
is important, as it can potentially slow the progression of this
devastating disease,” said Fosca De Iorio, Vice President,
International Medical Affairs at Vertex. “Today’s news provides
great hope that, if approved, a treatment option will be available
for the first time for about 300 young children with CF who have
two copies of the F508del mutation.”
In the European Union, ORKAMBI® (lumacaftor/ivacaftor) is
already approved for the treatment of people with CF who have two
copies of the F508del mutation, ages 2 years and above.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease
affecting more than 88,000 people globally. CF is a progressive,
multi-organ disease that affects the lungs, liver, pancreas, GI
tract, sinuses, sweat glands 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, and these mutations
can be identified by a genetic test. While there are many different
types of CFTR mutations that can cause the disease, the vast
majority of people with CF have at least one F508del mutation. CFTR
mutations lead to CF by causing CFTR protein to be defective or by
leading to a shortage or absence of CFTR protein 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, chronic lung infections and
progressive lung damage that eventually leads to death for many
patients. The median age of death is in the early 30s.
About ORKAMBI® (lumacaftor/ivacaftor)
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.
ORKAMBI® (lumacaftor/ivacaftor) is an oral medicine that is a
combination of lumacaftor and ivacaftor. Lumacaftor 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. Ivacaftor, which is known as a CFTR potentiator, is
designed to facilitate the ability of CFTR proteins to transport
salt and water across the cell membrane. The combined actions of
lumacaftor and ivacaftor help 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
clinical pipeline of investigational small molecule, mRNA, cell and
genetic therapies (including gene editing) in other serious
diseases where it has deep insight into causal human biology,
including sickle cell disease, beta thalassemia, APOL1-mediated
kidney disease, acute and neuropathic pain, type 1 diabetes and
alpha-1 antitrypsin deficiency.
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 13 consecutive years on Science magazine's Top
Employers list and one of Fortune’s 100 Best Companies to Work For.
For company updates and to learn more about Vertex's history of
innovation, visit www.vrtx.com or follow us on Twitter, LinkedIn
and YouTube.
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 Fosca De Iorio in
this press release and statements regarding the eligible patient
population for ORKAMBI®, our expectations for the number of
patients newly eligible for ORKAMBI®, and statements regarding the
potential benefits of ORKAMBI®. 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 factors that could cause actual events or results to
differ materially from those indicated by such forward-looking
statements. Those risks and uncertainties include risks listed
under the heading “Risk Factors” in Vertex's annual report and in
subsequent filings filed with the Securities and Exchange
Commission and available through the company's website at
www.vrtx.com and www.sec.gov. 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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