-A new treatment option for patients with two
copies of the F508del mutation, the most common mutation in cystic
fibrosis-
-First medicine in Australia to treat the
underlying cause of cystic fibrosis in patients who have certain
mutations that result in residual CFTR function-
Vertex Pharmaceuticals Incorporated today announced that the
Therapeutic Goods Administration (TGA) of Australia has granted
registration to SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for
the treatment of people with cystic fibrosis (CF) aged 12 years and
older who are homozygous for the F508del mutation or who have at
least one mutation in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor
based on in vitro data and/or clinical evidence. Mutations in the
CFTR gene that produce CFTR protein responsive to SYMDEKO® include
F508del and mutations in which the CFTR protein shows residual
function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L,
S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, 3849+10kbC→T, E56K,
R74W, D110E, D110H, E193K, E831X, F1052V, K1060T, A1067T, F1074L
and D1270N. SYMDEKO® will be considered for Australian
reimbursement for eligible CF patients aged 12 years and older at
the March meeting of the Pharmaceutical Benefits Advisory
Committee.
“We are delighted that the Therapeutic Goods Administration in
Australia recognized the safety profile and efficacy of SYMDEKO®.
This approval brings us one step closer to our future goal of
bringing treatment to all people living with CF,” said Reshma
Kewalramani, M.D., Executive Vice President and Chief Medical
Officer at Vertex. “This new medicine is an especially important
treatment option for patients with residual function mutations and
those with two copies of the F508del mutation who either never
started or discontinued ORKAMBI® (lumacaftor/ivacaftor).”
The TGA’s decision is 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 (≥10% incidence) experienced by patients
who received tezacaftor/ivacaftor in combination with ivacaftor in
pooled, placebo-controlled Phase 3 studies were headache and
nasopharyngitis.
Tezacaftor/ivacaftor in combination with ivacaftor was approved
by the U.S. Food and Drug Administration (FDA) in February 2018, by
Health Canada in June 2018 and by the European Commission in
October 2018.
About Cystic Fibrosis
Cystic Fibrosis (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 cystic fibrosis
transmembrane conductance regulator (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 SYMDEKO® (tezacaftor/ivacaftor and
ivacaftor)
Some mutations result in CFTR protein that is not processed or
folded normally within the cell, and that generally does not reach
the cell surface. SYMDEKO is a combination of tezacaftor and
ivacaftor. 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.
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR
SYMDEKO® (tezacaftor/ivacaftor and ivacaftor)
tablets:
SYMDEKO is a prescription medicine used for the treatment of
cystic fibrosis (CF) in patients aged 12 years and older who have
two copies of the F508del mutation, or who have at least one
mutation in the CF gene that is responsive to treatment with
SYMDEKO. Patients should talk to their doctor to learn if they have
an indicated CF gene mutation. It is not known if SYMDEKO is safe
and effective in children under 12 years of age.
Patients should not take SYMDEKO if they take certain
medicines or herbal supplements such as: the antibiotics
rifampin or rifabutin; seizure medicines such as phenobarbital,
carbamazepine, or phenytoin; St. John’s wort.
Before taking SYMDEKO, patients should tell their doctor if
they: have or have had liver problems; have kidney problems;
are pregnant or plan to become pregnant because it is not known if
SYMDEKO will harm an unborn baby; are breastfeeding or planning to
breastfeed because it is not known if SYMDEKO passes into breast
milk.
SYMDEKO may affect the way other medicines work, and other
medicines may affect how SYMDEKO works. Therefore, the dose of
SYMDEKO may need to be adjusted when taken with certain medicines.
Patients should especially tell their doctor if they take
antifungal medicines such as ketoconazole, itraconazole,
posaconazole, voriconazole, or fluconazole; or antibiotics such as
telithromycin, clarithromycin, or erythromycin.
SYMDEKO may cause dizziness in some people who take it.
Patients should not drive a car, use machinery, or do anything that
requires alertness until they know how SYMDEKO affects them.
Patients should avoid food or drink that contains
grapefruit or Seville oranges while they are taking SYMDEKO.
SYMDEKO can cause serious side effects, including:
High liver enzymes in the blood, which have been reported
in people treated with SYMDEKO or treated with ivacaftor alone. The
patient’s doctor will do blood tests to check their liver before
they start SYMDEKO, every 3 months during the first year of taking
SYMDEKO, and every year while taking SYMDEKO. Patients should call
their doctor right away if they have any of the following symptoms
of liver problems: pain or discomfort in the upper right stomach
(abdominal) area; yellowing of the skin or the white part of the
eyes; loss of appetite; nausea or vomiting; dark, amber-colored
urine.
Abnormality of the eye lens (cataract) in some children
and adolescents treated with SYMDEKO or with ivacaftor alone. If
the patient is a child or adolescent, their doctor should perform
eye examinations before and during treatment with SYMDEKO to look
for cataracts.
The most common side effects of SYMDEKO include headache,
nausea, sinus congestion, and dizziness.
These are not all the possible side effects of SYMDEKO.
Please click here to see the full U.S.
Prescribing Information for SYMDEKO (tezacaftor/ivacaftor and
ivacaftor) tablets.
About EVOLVE and EXPAND
Data 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 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, 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 nine years in a row. For additional
information and the latest updates from the company, please visit
www.vrtx.com.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995,
including, without limitation, the expected March meeting of the
Pharmaceuticals Benefits Advisory Committee and the quote by Dr.
Kewalramani in the second paragraph. While Vertex believes the
forward-looking statements contained in this press release are
accurate, these forward-looking statements represent the company's
beliefs only as of the date of this press release and 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, that data from the company's development programs may not
support registration or further development of its compounds due to
safety, efficacy or other reasons, and other risks listed under
Risk Factors in Vertex's annual report and quarterly reports filed
with the Securities and Exchange Commission and available through
the company'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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