-Study met primary endpoint with a
statistically significant improvement in absolute change in lung
clearance index (LCI2.5) through 8 weeks of tezacaftor/ivacaftor
treatment-
-Tezacaftor in combination with ivacaftor was
generally well tolerated and safety data were consistent with
previous studies-
-Data support a submission to the European
Medicines Agency in the second half of 2019-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the results of a Phase 3 study conducted in Europe and
Australia of tezacaftor in combination with ivacaftor in children
aged 6 through 11 years with cystic fibrosis (CF) who have either
two copies of the F508del mutation or one copy of the F508del
mutation and one residual function mutation. The study met its
primary endpoint of absolute change in lung clearance index
(LCI2.5) through 8 weeks of treatment, demonstrating a
statistically significant improvement in LCI2.5 among patients
treated with tezacaftor/ivacaftor. The regimen was generally well
tolerated and safety data were consistent with those observed in
previous studies with tezacaftor/ivacaftor. This efficacy study was
designed to support a submission to the European Medicines Agency
(EMA) to extend the indication of tezacaftor/ivacaftor in this
patient population. Vertex plans to submit the application in the
second half of 2019. In late 2018, Vertex submitted an sNDA to the
U.S. Food and Drug Administration (FDA) for tezacaftor/ivacaftor
based on a previously completed Phase 3 safety study in children
ages 6 through 11 years of age conducted in the U.S. and
Canada.
“These data mark an important milestone in our efforts to expand
treatment options for patients living with CF,” said Reshma
Kewalramani, M.D., Executive Vice President and Chief Medical
Officer at Vertex. “We plan to submit an indication extension to
the EMA in the second half of 2019, bringing us a step closer to
potentially providing more children with a treatment option that
addresses the underlying cause of the disease.”
Summary of Key Data
The data announced today are from a Phase 3, randomized,
double-blind, parallel-group study to evaluate the efficacy and
safety of tezacaftor in combination with ivacaftor in children ages
6 through 11 who have either two copies of the F508del mutation or
one copy of the F508del mutation and one residual function
mutation. Subjects were randomized 4:1 based on their genotype to
tezacaftor/ivacaftor versus a blinding arm (placebo for those with
two copies of F508del; ivacaftor for those with one copy of F508del
mutation and one residual function mutation). The study randomized
and treated 54 subjects with TEZ/IVA, 10 with placebo, and 3 with
ivacaftor.
The primary endpoint of the study was the within-group absolute
change in lung clearance index (LCI2.5) from baseline through Week
8 in patients treated with tezacaftor/ivacaftor. LCI2.5 measures
the efficiency of ventilation in the lungs by quantifying how many
standard lung volumes it takes to reduce exhaled nitrogen to 2.5
percent of its starting value when breathing pure oxygen. LCI is
considered a more sensitive measure to detect early lung disease
than forced expiratory volume in one second (FEV1). Higher LCI
scores indicate poorer lung function. To participate in the study,
children at an initial screening visit had to have an LCI2.5 ≥7.5,
which is considered the cutoff for abnormal gas exchange. In the
study, 54 children that were treated with tezacaftor/ivacaftor
experienced a mean within-group absolute improvement in LCI2.5 of
-0.51 through 8 weeks (p < 0.0001).
Overall, safety data were similar to those observed in previous
studies of tezacaftor/ivacaftor. The most common adverse events (≥
10%) among those patients receiving tezacaftor/ivacaftor were
cough, headache, and productive cough. No serious adverse events or
adverse events leading to treatment discontinuation or interruption
were observed.
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 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.
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), SYMDEKO®
(tezacaftor/ivacaftor and ivacaftor), VX-659 and VX-445 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,
including, without limitation, the statements in the first
paragraph of the press release and 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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Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:mediainfo@vrtx.comorNorth America: +
1-617-341-6992orEurope & Australia: + 44 20 3204 5275
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