Market Overview

Health Canada Grants Market Authorization for KALYDECO® (ivacaftor) in Children Ages 12 to <24 months with Certain Mutations in the CFTR Gene


-KALYDECO is the first and only approved medicine in Canada to treat
the underlying cause of cystic fibrosis in these young patients-

Pharmaceuticals Incorporated
(NASDAQ:VRTX) today announced that
Health Canada has granted Market Authorization for KALYDECO® (ivacaftor)
to include use in children with cystic fibrosis (CF) ages 12 to <24
months who have one of the following mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D,
G178R, G551S, S1251N, S1255P, S549N or S549R.

"We believe it is important to treat the underlying cause of cystic
fibrosis as early as possible, and are pleased that parents and
physicians now have a medicine to treat the underlying cause of CF in
indicated patients as young as one year of age," said Reshma
Kewalramani, M.D., Executive Vice President and Chief Medical Officer at
Vertex. "We look forward to working with the Canadian health officials
and provinces to achieve rapid access to KALYDECO for this small, but
important group of young children."

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 a gating mutation in the CFTR gene. The study 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.

KALYDECO was previously approved for expanded use in patients aged 12 to
<24 months by the U.S. Food and Drug Administration (FDA) in August of
2018 and by the European Commission in November of the same year.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 75,000 people in North America, Europe and Australia,
including 4,200 people in Canada.

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 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). 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. Two
patients had elevated liver enzymes greater than eight times the upper
limit of normal, but continued to receive KALYDECO 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 KALYDECO, 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. 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, KALYDECO 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. KALYDECO is available as 150
mg tablets for adults and pediatric patients age 6 years and older, and
is taken with fat-containing food. It is also available as 50 mg and 75
mg granules in pediatric ages 12 months to less than 6 years and is
administered with soft-food or liquid with fat-containing food.

People with CF who have specific mutations in the CFTR gene are
currently benefiting from KALYDECO in 27 different countries across
North America, Europe and Australia.


KALYDECO (ivacaftor) is a prescription medicine
used for the treatment of cystic fibrosis (CF) in patients age 12 months
and older who have at least one mutation in their CF gene that is
responsive to KALYDECO. Patients should talk to their doctor to learn if
they have an indicated CF gene mutation. It is not known if KALYDECO is
safe and effective in children under 12 months of age.

Patients should not take KALYDECO if they take certain medicines or
herbal supplements, such as:
the antibiotics rifampin or rifabutin;
seizure medications such as phenobarbital, carbamazepine, or phenytoin;
or St. John's wort.

Before taking KALYDECO, patients should tell their doctor if they:
have liver or kidney problems; drink grapefruit juice, or eat grapefruit
or Seville oranges; are pregnant or plan to become pregnant because it
is not known if KALYDECO will harm an unborn baby; and are breastfeeding
or planning to breastfeed because is not known if KALYDECO passes into
breast milk.

KALYDECO may affect the way other medicines work, and other medicines
may affect how KALYDECO works.
Therefore the dose of KALYDECO may
need to be adjusted when taken with certain medications. Patients should
especially tell their doctor if they take antifungal medications such as
ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole;
or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO can cause dizziness in some people who take it. Patients should
not drive a car, use machinery, or do anything that needs them to be
alert until they know how KALYDECO affects them. Patients should avoid
food containing grapefruit or Seville oranges while taking KALYDECO.

KALYDECO can cause serious side effects.

High liver enzymes in the blood have been reported in patients
receiving KALYDECO.
The patient's doctor will do blood tests to
check their liver before starting KALYDECO, every 3 months during the
first year of taking KALYDECO, and every year while taking KALYDECO. For
patients who have had high liver enzymes in the past, the doctor may do
blood tests to check the liver more often. 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 their skin or the white part of their eyes; loss of
appetite; nausea or vomiting; or dark, amber-colored urine.

Abnormality of the eye lens (cataract) has been noted in some
children and adolescents receiving KALYDECO. The patient's doctor should
perform eye examinations prior to and during treatment with KALYDECO to
look for cataracts. The most common side effects include headache; upper
respiratory tract infection (common cold), which includes sore throat,
nasal or sinus congestion, and runny nose; stomach (abdominal) pain;
diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO. Please click here
to see the full U.S. Prescribing Information for KALYDECO.

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

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, as amended,
including the statements by Dr. Kewalramani in the second paragraph.
While the company 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 regulatory authorities
may not approve, or approve on a timely basis, the company's drug
candidates due to safety, efficacy or other reasons, and the other risks
listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and available
through Vertex's website at
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.


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