Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) to Treat Patients with Cystic Fibrosis Aged 12 to <24 months with Certain Mutations in the CFTR Gene

LONDON–(BUSINESS WIRE)–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 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)
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 nine 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 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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