Vertex Receives European CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fib...
November 16 2018 - 7:57AM
Business Wire
- If approved, lumacaftor/ivacaftor will be the
first medicine in Europe to treat the underlying cause of cystic
fibrosis for approximately 1,500 young children with two copies of
the F508del mutation -
Vertex Pharmaceuticals (Europe) Limited today announced that the
European Medicines Agency’s (EMA) Committee for Medicinal Products
for Human Use (CHMP) adopted a positive opinion for ORKAMBI®
(lumacaftor/ivacaftor) for the treatment of people 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.
If the European Commission issues a favorable adoption of the
EMA CHMP opinion for the extension of indication,
lumacaftor/ivacaftor will be the first and only medicine approved
in Europe to treat the underlying cause of CF for patients aged 2
to 5 years old who have two copies of the F508del mutation.
“Cystic fibrosis is a chronic, progressive disease and it is
important to treat early to ensure the best possible outcomes for
patients,” said Reshma Kewalramani, MD, Executive Vice President,
Global Medicines Development and Medical Affairs and Chief Medical
Officer at Vertex. “Today’s announcement brings us one step closer
to providing more young children with a treatment that addresses
the underlying cause of their disease. It also marks another
important milestone on our path towards our goal of treating every
person with this rare and life-limiting disease.”
The submission was supported by 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 Europe 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 CHMP
positive opinion 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 third
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, that Vertex could experience unforeseen delays
in obtaining the European Commission favorable adoption of the EMA
CHMP opinion for the extension of indication 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/20181116005219/en/
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1-617-341-6108orEric Rojas, +1-617-961-7205orZach Barber,
+1-617-341-6470 orMedia: mediainfo@vrtx.comorNorth
America:Sarah D'Souza, + 1-857-329-8495orEurope &
Australia:Marie von Seyfried, + 44 7933 500887
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From Apr 2024 to May 2024
Vertex Pharmaceuticals (NASDAQ:VRTX)
Historical Stock Chart
From May 2023 to May 2024