Market Overview

Vertex Receives a Positive PBAC Recommendation for Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) to Treat Australians Ages Six and Over with Cystic Fibrosis and Two Copies of the F508del Mutation


- PBAC recommendation moves approximately 1,300 patients in Australia
closer to access to lumacaftor/ivacaftor and Vertex is now working with
the Australian Government to finalize a reimbursement agreement as soon
as possible -

Vertex Pharmaceuticals (Europe) Limited announces that it has received
the Pharmaceutical Benefits Advisory Committee's (PBAC) recommendation
for ORKAMBI® (lumacaftor/ivacaftor) to be listed on the
Pharmaceutical Benefits Scheme (PBS) in Australia for people ages six
and over with cystic fibrosis (CF) who have two copies of the F508del mutation
in the cystic fibrosis transmembrane conductance regulator (CFTR)
gene. Lumacaftor/ivacaftor is the first medicine to treat the underlying
cause of CF in people ages six and older who have two copies of the F508del

"We're very pleased that lumacaftor/ivacaftor received a positive PBAC
recommendation for CF patients in Australia, which brings those who have
long been waiting closer to receiving this important medicine," said
Simon Bedson, International General Manager at Vertex. "We welcome the
PBAC decision and are now working with the Australian Government to
finalize the agreement as quickly as possible to make
lumacaftor/ivacaftor available to patients."

Many thousands of patients worldwide are already receiving
lumacaftor/ivacaftor in countries where it is reimbursed including
Austria, Denmark, Germany, Ireland, Italy, Sweden, the Netherlands and
the U.S.

Vertex remains steadfast in its commitment to ensuring people living
with CF have rapid access to its medicines.

About CF

Cystic fibrosis is a rare, life-shortening genetic disease affecting
approximately 75,000 people including countries within North America and
Europe, as well as 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 ORKAMBI® (lumacaftor/ivacaftor) and
the F508del mutation

In people with two copies of the F508del mutation, the CFTR
protein is not processed and trafficked normally within the cell,
resulting in little-to-no CFTR protein at the cell surface. Patients
with two copies of the F508del mutation are easily identified by
a simple genetic test.

ORKAMBI® is a combination of lumacaftor, which is designed to
increase the amount of mature protein at the cell surface by targeting
the processing and trafficking defect of the F508del-CFTR protein, and
ivacaftor, which is designed to enhance the function of the CFTR protein
once it reaches the cell surface. Lumacaftor/ivacaftor is available as
tablets and is typically taken twice per day.

For complete product information, please see the Summary of Product
Characteristics that can be found on

About the Australian Pharmaceutical Benefits Advisory Committee (PBAC)

The PBAC is an independent, expert statutory body which reviews
submissions from pharmaceutical companies regarding new medicines to be
considered for subsidy within the Pharmaceutical Benefits Scheme (PBS).
The PBAC makes recommendations to the Government as to whether or not a
medicine should be subsidized and the Health Minister cannot add a
medicine to the PBS without prior recommendation from the PBAC.

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 Brazil. 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, Mr. Bedson'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, that Vertex could experience
unforeseen delays in reaching an agreement with the Australian
government 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


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