- In Europe, there are approximately 3,400
children ages 6-11 who have two copies of the F508del mutation
-
- Existing reimbursement agreements in
countries like Ireland will enable rapid access to ORKAMBI;
country-by-country reimbursement processes will now begin in other
countries-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the European Commission has granted extension of the
Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), the
first medicine to treat the underlying cause of cystic fibrosis
(CF) in people with two copies of the F508del mutation, to include
children ages 6 through 11. In Europe, there are approximately
3,400 children ages 6 through 11 with two copies of this mutation.
Existing reimbursement agreements in countries such as Ireland will
enable rapid access to ORKAMBI for these children. In other
countries across the European Union, Vertex will now begin the
country-by-country reimbursement process.
“The innovative long-term agreements we have reached in
countries like Ireland will enable eligible children to have rapid
access to ORKAMBI,” said Simon Bedson, Senior Vice President and
International General Manager at Vertex. “Where these agreements
are not in place, Vertex is committed to working with local
authorities so those who could benefit from this medicine are able
to do so as quickly as possible.”
The European Commission’s decision is based on data from two
Phase 3 studies of ORKAMBI in children with CF ages 6 through 11
who have two copies of the F508del mutation. In 2017, The Lancet
Respiratory published 24-week data from one of these studies, which
demonstrated statistically significant improvements in lung
function (as assessed by the absolute change in lung clearance
index, or LCI2.5, and predicted forced expiratory volume in one
second, or ppFEV1) among children treated with ORKAMBI compared to
placebo. Improvements in body mass index (BMI) and the Cystic
Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
were also observed, although they were not statistically
significant. Safety data were similar to those observed in an
earlier Phase 3 open-label safety study in children ages 6 through
11. The most common adverse events that occurred more frequently
among those receiving ORKAMBI compared to placebo were infective
pulmonary exacerbation, productive cough, nasal congestion,
oropharyngeal pain, abdominal pain upper, headache, upper
respiratory tract infection and sputum increased.
“A principal goal of treating CF is slowing the progressive lung
damage caused by this life-shortening genetic disease while
improving health in the short term,” said Professor Marcus Mall,
M.D., Director of the Division of Pediatric Pulmonology &
Allergology and the Cystic Fibrosis Center at the Heidelberg
University Hospital, Germany. “Studies of ORKAMBI in children ages
6 through 11 have shown improvements in clinically relevant
outcomes, like lung function and weight gain.”
About Cystic Fibrosis (CF)
CF is a rare, life-shortening genetic disease affecting
approximately 75,000 people across 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 protein 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 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
mid-to-late 20s.
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.
Patients with two copies of the F508del mutation are easily
identified by a simple genetic test.
ORKAMBI 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. ORKAMBI is
available as tablets and is typically taken twice per day.
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 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 and Australia. 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 eight years in a row.
For additional information and the latest updates from the company,
please visit www.vrtx.com.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and
development affiliate of the Cystic Fibrosis Foundation.
KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor),
tezacaftor, VX-440, VX-152, VX-659 and VX-445 were discovered by
Vertex as part of this collaboration.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements, as
defined in the Private Securities Litigation Reform Act of 1995, as
amended, including the quotes in the second and fourth paragraphs
of this press release and statements regarding the timing of access
to ORKAMBI for patients 6 through 11 and the country-by-country
reimbursement approval process. While the company 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
patients 6 through 11 in Europe and the other risks listed under
Risk Factors in Vertex's annual report and quarterly reports filed
with the Securities and Exchange Commission and available through
Vertex's website at www.vrtx.com. 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 IncorporatedInvestors:Michael
Partridge, 617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comorEurope & Australia:Rebecca
Hunt, +44 7718 962 690orNorth America:Megan Goulart, +
1-617-341-6992
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