If approved, ivacaftor will be the first and
only medicine to treat the underlying cause of cystic fibrosis for
these young children
Vertex Pharmaceuticals (Europe) Limited today announces that the
European Medicines Agency’s (EMA) Committee for Medicinal Products
for Human Use (CHMP) adopted a positive opinion 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.
If the European Commission issues a favorable adoption of the
EMA CHMP opinion for the extension of indication, ivacaftor will be
the first and only medicine approved in Europe to treat the
underlying cause of CF in patients aged 12 to <24 months, who
have specific mutations in the CFTR gene.
“Cystic fibrosis is a chronic, progressive disease that is
present at birth, with symptoms often occurring in infancy, so
early treatment is crucial to deliver the best possible outcomes
for patients,” said Reshma Kewalramani, MD, Executive Vice
President, Global Medicines Development and Medical Affairs and
Chief Medical Officer at Vertex. “Today’s announcement marks an
important step towards allowing young CF patients to benefit from
treatment at an early stage of their disease, and brings us one
step closer to our goal of treating all people living with CF.”
The submission was supported by 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 CF
Cystic 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 Study
The 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)
KALYDECO® (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 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 eight years in a row.
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, Dr. Kewalramani’s statement in the
third 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, that Vertex could experience unforeseen delays
in obtaining marketing approval from the European Commission 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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