Vertex Announces Reimbursement Agreement in Italy for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Under...
July 13 2017 - 4:00AM
Business Wire
- Effective immediately, agreement enables
hundreds of people in Italy to access this important medicine -
- Recent pricing and reimbursement agreements
have enabled broad access to ORKAMBI for thousands of eligible
patients in multiple European countries; negotiations continue in a
number of other countries, including France and the United Kingdom
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the Italian Medicines Agency (Agenzia Italiana del
Farmaco, or AIFA) has agreed to reimburse ORKAMBI®
(lumacaftor/ivacaftor), the first medicine to treat the underlying
cause of cystic fibrosis (CF) in people ages 12 and older who have
two copies of the F508del mutation in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. The agreement is
published online in the Italian Official Gazette. Regional
authorities will now begin implementation to provide the hundreds
of eligible patients in Italy access to this important medicine.
Recent European pricing and reimbursement agreements have enabled
broad access to ORKAMBI for thousands of eligible patients in
Austria, Denmark, Germany, Ireland, Italy and Luxembourg.
Negotiations continue in a number of other countries where CF is
prevalent, including France and the United Kingdom.
“We are pleased to have reached this agreement on behalf of CF
patients in Italy who have been waiting for this important
medicine,” said Simon Bedson, Senior Vice President and
International General Manager at Vertex. “We continue negotiations
with other countries including France and the United Kingdom, and
we encourage these national health authorities and governments to
work quickly with us to achieve reimbursement for all patients who
may benefit.”
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 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), ORKAMBI® (lumacaftor/ivacaftor) and tezacaftor 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.comorEurope & Australia:Megan
Goulart, +44 20 3204 5275orNorth America:David Whitrap, +1 617 961
5093
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