Market Overview

Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation


- In Europe, there are approximately 3,400 children ages 6-11 who
have two copies of the F508del mutation -

- Existing reimbursement agreements in countries like Ireland will
enable rapid access to ORKAMBI; country-by-country reimbursement
processes will now begin in other countries-

Pharmaceuticals Incorporated
(NASDAQ:VRTX) today announced that the
European Commission has granted extension of the Marketing Authorization
for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to
treat the underlying cause of cystic fibrosis (CF) in people with two
copies of the F508del mutation, to include children ages 6
through 11. In Europe, there are approximately 3,400 children ages 6
through 11 with two copies of this mutation. Existing reimbursement
agreements in countries such as Ireland will enable rapid access to
ORKAMBI for these children. In other countries across the European
Union, Vertex will now begin the country-by-country reimbursement

"The innovative long-term agreements we have reached in countries like
Ireland will enable eligible children to have rapid access to ORKAMBI,"
said Simon Bedson, Senior Vice President and International General
Manager at Vertex. "Where these agreements are not in place, Vertex is
committed to working with local authorities so those who could benefit
from this medicine are able to do so as quickly as possible."

The European Commission's decision is based on data from two Phase 3
studies of ORKAMBI in children with CF ages 6 through 11 who have two
copies of the F508del mutation. In 2017, The Lancet Respiratory
published 24-week data from one of these studies, which demonstrated
statistically significant improvements in lung function (as assessed by
the absolute change in lung clearance index, or LCI2.5, and
predicted forced expiratory volume in one second, or ppFEV1)
among children treated with ORKAMBI compared to placebo. Improvements in
body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised
(CFQ-R) respiratory domain score were also observed, although they were
not statistically significant. Safety data were similar to those
observed in an earlier Phase 3 open-label safety study in children ages
6 through 11. The most common adverse events that occurred more
frequently among those receiving ORKAMBI compared to placebo were
infective pulmonary exacerbation, productive cough, nasal congestion,
oropharyngeal pain, abdominal pain upper, headache, upper respiratory
tract infection and sputum increased.

"A principal goal of treating CF is slowing the progressive lung damage
caused by this life-shortening genetic disease while improving health in
the short term," said Professor Marcus Mall, M.D., Director of the
Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis
Center at the Heidelberg University Hospital, Germany. "Studies of
ORKAMBI in children ages 6 through 11 have shown improvements in
clinically relevant outcomes, like lung function and weight gain."

About Cystic Fibrosis (CF)

CF is a rare, life-shortening genetic disease affecting approximately
75,000 people across 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 protein 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 ORKAMBI® (lumacaftor/ivacaftor)

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. ORKAMBI 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 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

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), tezacaftor, VX-440, VX-152,
VX-659 and VX-445 were discovered by Vertex as part of this

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 quotes in the second and fourth paragraphs of this press
release and statements regarding the timing of access to ORKAMBI for
patients 6 through 11 and the country-by-country reimbursement approval
process. 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, risks related to
commercializing ORKAMBI for patients 6 through 11 in Europe 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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