Ivacaftor is the first and only approved
medicine in Europe to treat the underlying cause of cystic fibrosis
in these young patients
Vertex Pharmaceuticals (Europe) Limited today announced that the
European Commission has granted approval of the label extension for
KALYDECO® (ivacaftor) to include the treatment of people with
cystic fibrosis (CF) aged 12 to <24 months who have at least one
of the following nine mutations in their cystic fibrosis
transmembrane conductance regulator (CFTR) gene: G551D, G1244E,
G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
“For the first time, EU physicians can now treat the underlying
cause of CF earlier than ever, helping to improve clinical outcomes
in children as young as 12 months,” said Reshma Kewalramani, MD,
Executive Vice President, Global Medicines Development and Medical
Affairs and Chief Medical Officer at Vertex. “We look forward to
supporting additional research on the potential benefit of early
intervention with our medicines, with the goal of bringing
treatment to all people living with CF.”
The label update is based on data from the ongoing Phase 3
open-label safety study (ARRIVAL) of children with CF aged 12 to
<24 months who have one of 10 mutations in the CFTR gene that
demonstrated a safety profile consistent with that observed in
previous Phase 3 studies of older children and adults, and
improvements in sweat chloride, a key secondary efficacy
endpoint.
Ivacaftor is already approved in Europe for the treatment of CF
in patients aged two years and older who have one of the nine
following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R,
G551S, S1251N, S1255P, S549N or S549R. It is also approved for the
treatment of CF in patients aged 18 years and older who have an
R117H mutation in the CFTR gene.
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 the ARRIVAL StudyThe ARRIVAL study is an ongoing
Phase 3 open-label safety study of 25 children with CF aged 12 to
<24 months who have one of 10 mutations in the CFTR gene (G551D,
G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or
R117H; patients with the R117H mutation were eligible to enroll in
regions where ivacaftor is approved for use in patients 2 through 5
years of age with an R117H mutation). The study demonstrated a
safety profile consistent with that observed in previous Phase 3
studies of older children and adults; most adverse events were mild
or moderate in severity, and no patient discontinued due to adverse
events. Treatment was interrupted in two patients who had elevated
liver enzymes greater than eight times the upper limit of normal,
but continued to receive ivacaftor after a dose interruption. The
most common adverse events (≥30%) were cough (74%), pyrexia (37%),
elevated aspartate aminotransferase (37%), elevated alanine
aminotransferase (32%) and runny nose (32%). Four serious adverse
events were observed in two patients.
Mean baseline sweat chloride for the children in this study was
104.1 mmol/L (n=14). Following 24 weeks of treatment with
ivacaftor, the mean sweat chloride level was 33.8 mmol/L (n=14). In
the 10 subjects with paired sweat chloride samples at baseline and
week 24, there was a mean absolute change of -73.5 mmol/L. Sweat
chloride is used as a tool to diagnose infants with CF, where
levels greater than or equal to 60 mmol/L indicate that CF is
likely, levels of 30-59 mmol/L indicate CF is possible and levels
less than 30 indicate that CF is unlikely. These data were
presented at the 41st European Cystic Fibrosis Society (ECFS)
Conference in June 2018 and published in The Lancet Respiratory
Medicine (Volume 6, No 7, July 2018).
About KALYDECO® (ivacaftor)Ivacaftor is the
first medicine to treat the underlying cause of CF in people with
specific mutations in the CFTR gene. Known as a CFTR potentiator,
ivacaftor is an oral medicine designed to keep CFTR proteins at the
cell surface open longer to improve the transport of salt and water
across the cell membrane, which helps hydrate and clear mucus from
the airways.
People with CF who have specific mutations in the CFTR gene are
currently benefiting from ivacaftor in countries across North
America, Europe and in Australia.
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 nine 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, including,
without limitation, Dr. Kewalramani’s statement in the second
paragraph of this press release. While Vertex 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 KALYDECO® for
people with cystic fibrosis aged 12 to <24 months 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,
+1-617-341-6470 orMedia: mediainfo@vrtx.comorNorth
America:Sarah D'Souza, + 1-617-341-6341orEurope &
Australia:Marie von Seyfried, + 44 7933 500887
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