Vertex Announces German Reimbursement Agreement for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Underly...
December 19 2016 - 4:30AM
Business Wire
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today
announced it has reached a pricing and reimbursement agreement for
ORKAMBI® (lumacaftor/ivacaftor) with the German Federal Association
of the Statutory Health Insurances (GKV-SV). ORKAMBI is 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 CF transmembrane conductance regulator (CFTR) gene.
CF is a rare and life-shortening genetic disease caused by a
defective or missing CFTR protein resulting from a mutation in the
CFTR gene. The disease is present from birth and causes chronic
lung infections and progressive damage to a number of organs
throughout the body.
Today’s announcement follows a comprehensive benefit assessment
of the medicine by the German Federal Joint Committee (G-BA), which
recognized the “considerable additional benefit” of ORKAMBI for
people with CF who have two copies of the F508del mutation. ORKAMBI
has been available to eligible patients in Germany since it was
granted marketing authorization from the European Commission in
November 2015, and the reimbursement agreement announced today took
effect on December 16, 2016.
“Cystic fibrosis is a progressive disease, meaning that with
each passing day, lung function can deteriorate and patients get
sicker,” said David Gillen, M.D., Vice President of International
Medical Affairs at Vertex. “We are pleased the German authorities
approved ORKAMBI reimbursement for all eligible patients. The
reimbursed price recognizes the clinical value of ORKAMBI and the
need for Vertex’s continued investment in the research and
development of new medicines for the two out of three people with
CF still waiting for a treatment for the underlying cause of
their form of the disease.”
In addition to Germany, ORKAMBI is now available to eligible
people with CF in the United States, Austria, and France. Vertex
remains actively involved in reimbursement discussions in many
other countries to make ORKAMBI available to all people who can
benefit from this important medicine. Earlier this month,
ORKAMBI received the “Drug Discovery of the Year” award from the
British Pharmacological Society and the French “Prix Galien” award
for the most promising rare disease medicine in 2016.
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) 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 third 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,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470Media:Europe & Australia:Megan Goulart, +44
20 3204 5275orNorth America:Chris Stamm, +1
617-341-6992mediainfo@vrtx.com
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