Vertex Announces Long-Term Reimbursement Agreement with the Republic of Ireland for ORKAMBI® (lumacaftor/ivacaftor), KALYDEC...
02 June 2017 - 3:15AM
Business Wire
-Agreement provides access to ORKAMBI for
people who have two copies of the F508del mutation and expands
access to KALYDECO for all eligible patients-
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today
announced it has reached an agreement with the Health Service
Executive (HSE) in the Republic of Ireland to fund ORKAMBI®
(lumacaftor/ivacaftor) for all of the approximately 500 people in
Ireland with cystic fibrosis (CF) ages 12 and older who have two
copies of the F508del mutation. The agreement also expands access
to KALYDECO® (ivacaftor) for children ages 2 to 5 with any approved
gating mutation (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N,
S1255P and G1349D) and to people ages 18 and older who have an
R117H mutation. These reimbursements are effective immediately.
This innovative long-term agreement also enables rapid access for
people with these mutations if the labels of the existing medicines
are expanded to cover additional age groups and if new Vertex
medicines are approved for these populations.
“We are pleased that these additional Irish CF patients will
finally join the thousands of others around the world who are
already benefitting from ORKAMBI and KALYDECO,” said Simon Bedson,
Senior Vice President and International General Manager at Vertex.
“We thank the leaders in Ireland for working with us toward an
innovative reimbursement agreement that provides access to these
important medicines and also recognizes the need for Vertex’s
continued investment in the research and development of new
medicines for those people with CF, many of whom are still waiting
for a treatment for the underlying cause of the disease.”
CF is a rare and life-shortening genetic disease caused by a
defective or missing cystic fibrosis transmembrane conductance
regulator (CFTR) protein resulting from a mutation in the CFTR
gene. ORKAMBI and KALYDECO are the first two medicines that treat
the underlying cause of CF. Ireland has the highest rate of CF in
the world, with approximately one in 19 Irish people carrying a
disease-causing mutation in one copy of the CFTR gene.
In addition to Ireland, ORKAMBI is available to all eligible
patients in Austria, Denmark, France, Germany, Luxembourg and the
United States. People in 27 countries are benefitting from
KALYDECO. Vertex remains actively involved in additional
reimbursement discussions globally, with the goal of making these
transformative medicines available to all eligible patients as soon
as possible.
About ORKAMBI® (lumacaftor/ivacaftor) and the
F508del mutation
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 KALYDECO® (ivacaftor)
KALYDECO® (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 that aims to help the CFTR protein function more normally
once it reaches the cell surface, to help hydrate and clear mucus
from the airways.
For complete product information, please see the Summary of
Product Characteristics that can be found at www.ema.europa.eu.
About CF
CF 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 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 Vertex
Vertex is a global biotechnology company that aims to discover,
develop and commercialize innovative medicines so people with
serious diseases can lead better lives. In addition to our clinical
development programs focused on cystic fibrosis, Vertex has more
than a dozen ongoing research programs aimed at other serious and
life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research
and development sites and commercial offices in the United States,
Europe, Canada and Australia. For seven years in a row, Science
magazine has named Vertex one of its Top Employers in the life
sciences. 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) and ORKAMBI® (lumacaftor/ivacaftor) 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 quote in the second paragraph of this press
release and statements regarding 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 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, +1
617-341-6108orEric Rojas, +1 617-961-7205orZach Barber, +1
617-341-6470orMedia:mediainfo@vrtx.comEurope &
Australia:Megan Goulart, +44 20 3204 5275orNorth America:David
Whitrap, +1 617-961-5093
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