- A new treatment option for patients with two
copies of the F508del mutation, the most common mutation in cystic
fibrosis -
- First medicine in the EU to treat the CFTR
protein defect in patients who have one copy of the F508del
mutation and one copy of one of 14 mutations that result in
residual CFTR activity -
Vertex Pharmaceuticals (Europe) Limited, today announced that
the European Commission has granted Marketing Authorization for
SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with
ivacaftor (KALYDECO®) for the treatment of people with cystic
fibrosis (CF) aged 12 and older who either have two copies of the
F508del mutation in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene, or one copy of the F508del mutation and a
copy of one of the following 14 mutations in which the CFTR protein
shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G,
711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and
3849+10kbC→T. In addition, the European Medicines Agency’s
Committee for Orphan Medicinal Products recently recommended the
maintenance of orphan designation for tezacaftor/ivacaftor in
combination with ivacaftor.
“The authorization of tezacaftor/ivacaftor in combination with
ivacaftor is welcome news for European CF patients, their families
and everyone involved in their treatment and care. This new
medicine is especially important for patients with residual
function mutations and those who do not tolerate ORKAMBI®
(lumacaftor/ivacaftor),” said Harry Heijerman, Professor and Head
of Department of Pulmonology at University Medical Centre Utrecht,
The Netherlands.
The EU Marketing Authorization was based on results from two
pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New
England Journal of Medicine in November 2017. Results showed
treatment with tezacaftor/ivacaftor in combination with ivacaftor
provides benefits across different CF populations, including
statistically significant improvements in lung function, as
determined by absolute change from baseline in percent predicted
forced expiratory volume in one second (ppFEV1); with a generally
well tolerated safety profile and a lack of increased respiratory
adverse events compared to placebo. The improvements in lung
function showed a mean absolute change in ppFEV1 compared to
placebo of 4.0 percentage points (P<0.0001) and 6.8 percentage
points (P<0.0001) in EVOLVE and EXPAND respectively. The most
common adverse reactions experienced by patients who received
tezacaftor/ivacaftor in combination with ivacaftor in pooled,
placebo-controlled Phase 3 studies were headache and
nasopharyngitis.
“Today marks an important milestone for many CF patients in
Europe, including those who so far have had no available option to
treat the CFTR protein defect responsible for their disease,” said
Reshma Kewalramani, MD, Executive Vice President, Global Medicines
Development and Medical Affairs and Chief Medical Officer at
Vertex. “With today’s Marketing Authorization, we are rapidly
moving towards treating 90 percent of CF patients.”
Tezacaftor/ivacaftor in combination with ivacaftor was approved
by the U.S. Food and Drug Administration (FDA) in February 2018 and
by Health Canada in June 2018. It is marketed as SYMDEKO™ in the
U.S. and Canada.
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 tezacaftor/ivacaftor and ivacaftorSome mutations
result in CFTR protein that is not processed or folded normally
within the cell, and that generally does not reach the cell
surface. Tezacaftor is designed to address the trafficking and
processing defect of the CFTR protein to enable it to reach the
cell surface where ivacaftor can increase the amount of time the
protein stays open.
For complete product information, please see the Summary of
Product Characteristics that can be found on www.ema.europa.eu once
posted.
About EVOLVE and EXPANDData from the two Phase 3 studies
EVOLVE and EXPAND were published in the New England Journal of
Medicine in November 2017, the studies enrolled approximately 750
people with CF ages 12 and older with two copies of the F508del
mutation or with one F508del mutation and a second mutation
associated with residual CFTR activity. Across both studies,
patients treated with tezacaftor/ivacaftor in combination with
ivacaftor experienced statistically significant improvements in
lung function, as determined by absolute change from baseline in
ppFEV1. The treatment was generally well tolerated; the most common
adverse reactions (≥10%) experienced by patients who received
tezacaftor/ivacaftor with ivacaftor in the pooled,
placebo-controlled Phase 3 studies were headache (14% versus 12% on
placebo) and nasopharyngitis (12% versus 10% on placebo).
About orphan designation for medicinesOrphan designation
is granted by the European Medicines Agency’s Committee for Orphan
Medicinal Products to treatments which either address an existing
unmet need or can provide significant benefit for people with
life-threatening or chronically debilitating diseases, affecting a
small number of patients.
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 eight 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, as amended,
including the quotes in the second and fourth paragraphs of this
press release. 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 SYMKEVI 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. 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.comorNorth
America:Heather Nichols, + 1-617-341-6992orEurope &
Australia:Marie von Seyfried, + 44 7933 500887
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