Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI (lumacaftor/ivacaftor)

Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that
ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat
the underlying cause of cystic fibrosis (CF) in people with two copies
of the F508del mutation, ages six and older, will be reimbursed
in Sweden after concluding the three-party negotiations with TLV and the
county councils. Reimbursement is effective from July 1. The innovative,
long-term access agreement also provides a framework for the assessment
and access of our future CF medicines.

“We are delighted that people with CF in Sweden will join the thousands
of others around the world who are already benefitting from our CF
medicines,” said Simon Bedson, International General Manager at Vertex.
“We commend the Swedish authorities for partnering with us on an
innovative, long-term access agreement. In countries where Vertex
remains actively involved in reimbursement discussions, we encourage
these health authorities and governments to match the commitment to
innovation shown in Sweden to secure access for all patients who may

CF is a devastating rare disease that causes continuous damage to
multiple organs from birth. In the lungs, a build-up of sticky mucus
causes progressive and permanent damage, severe infections and
ultimately premature death. In addition to Sweden, countries where
lumacaftor/ivacaftor is available to all eligible patients include
Austria, Germany, Ireland, Italy, the Netherlands and the U.S.

About Cystic Fibrosis

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 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 www.ema.europa.eu.

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 and Australia. 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.

For additional information and the latest updates from the company,
please visit www.vrtx.com.


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