Vertex Announces European Commission Approval for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibr...
January 21 2019 - 4:00AM
Business Wire
Lumacaftor/ivacaftor is the first and only
approved medicine in Europe to treat the underlying cause of cystic
fibrosis for approximately 1,500 children aged 2 to 5 with two
copies of the F508del mutation
Vertex Pharmaceuticals (Europe) Limited today announced that the
European Commission has granted approval of the label extension for
ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with
cystic fibrosis (CF) aged 2 to 5 years old who have two copies of
the F508del mutation, the most common form of the disease.
“Today’s approval by the European Commission brings us one step
closer to our goal of bringing treatment to all people living with
CF,” said Reshma Kewalramani, MD, Executive Vice President, Global
Medicines Development and Medical Affairs and Chief Medical Officer
at Vertex. ‘‘By treating the underlying cause of disease early, we
can potentially modify its course and offer patients the chance of
improved outcomes.’’
The label update is based on data from a Phase 3 open-label
safety study in 60 patients that showed treatment with
lumacaftor/ivacaftor was generally well tolerated for 24 weeks,
with a safety profile in these pediatric patients generally
consistent with that in patients aged 6 years and older.
Lumacaftor/ivacaftor is already approved in the EU for the
treatment of CF in patients aged 6 and older who have two copies of
the F508del mutation.
About CFCystic fibrosis is a rare, life-shortening
genetic disease affecting approximately 75,000 people in North
America, Europe and Australia.
CF is caused by a defective or missing CFTR protein resulting
from mutations in the CFTR gene. Children must inherit two
defective CFTR genes — one from each parent — to have CF. There are
approximately 2,000 known mutations in the CFTR gene. Some of these
mutations, which can be determined by a genetic test, or genotyping
test, lead to CF by creating non-working or too few CFTR proteins
at the cell surface. The defective function or absence of CFTR
protein results in poor flow of salt and water into and out of the
cell in a number of organs. In the lungs, this leads to the
build-up 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
mid-to-late 20s.
About the Phase 3 open-label safety studyThis European
Commission approval is based on a Phase 3 open-label safety study
in 60 patients that showed treatment with lumacaftor/ivacaftor was
generally well tolerated for 24 weeks, with a safety profile
similar to that in patients aged 6 years and older. Improvements in
sweat chloride, a secondary endpoint, were observed at week 24
(mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95%
CI: -35.7, -27.6, n=49). Researchers also saw changes in key growth
parameters, which were also secondary endpoints in the study. The
most common adverse event (≥30% overall) was cough (63%); most
adverse events were mild or moderate in severity. Four patients
experienced serious adverse events (2 infective pulmonary
exacerbations of cystic fibrosis, 1 gastroenteritis viral, 1
constipation) and three patients discontinued treatment due to
elevated transaminases without concurrent elevations in total
bilirubin. These findings were presented at the 41st European
Cystic Fibrosis Society Conference in June 2018.
About ORKAMBI®
(lumacaftor/ivacaftor)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.
About VertexVertex is a global biotechnology company that
invests in scientific innovation to create transformative medicines
for people with serious and life-threatening diseases. In addition
to clinical development programs in CF, Vertex has more than a
dozen ongoing research programs focused on the underlying
mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is
now located in Boston's Innovation District. Today, the company has
research and development sites and commercial offices in the United
States, Europe, Canada, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including being named to Science magazine's Top Employers in
the life sciences ranking for nine years in a row.
Special Note Regarding Forward-looking StatementsThis
press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including,
without limitation, Dr. Kewalramani’s statement in the second
paragraph of this press release. While Vertex believes the
forward-looking statements contained in this press release are
accurate, 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,
among other things, risks related to commercializing ORKAMBI® for
people with cystic fibrosis aged 2 to 5 years old and the other
risks listed under Risk Factors in Vertex's annual report and
quarterly reports filed with the Securities and Exchange
Commission. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190121005119/en/
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1-617-341-6108orEric Rojas, +1-617-961-7205orZach Barber,
+1-617-341-6470 or
Media: mediainfo@vrtx.comorNorth America:Sarah D'Souza,
+1-617-341-6341orEurope & Australia:Patricia Dessert, +44 7543
237825
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Apr 2023 to Apr 2024