Market Overview

Vertex Announces Reimbursement Agreement in Australia for ORKAMBI® (lumacaftor/ivacaftor) for People with Cystic Fibrosis Ages Six Years and Older with Two Copies of the F508del Mutation

Share:

- Approximately 1,300 patients in Australia join the thousands of
patients worldwide who already have access to lumacaftor/ivacaftor -

- A pathway to access for future Vertex CF medicine,
tezacaftor/ivacaftor, has also been established -

Vertex Pharmaceuticals (Europe) Limited announces finalization of an
agreement with the Australian Government that allows for reimbursement
of ORKAMBI® (lumacaftor/ivacaftor) 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.
This agreement means lumacaftor/ivacaftor will be listed on the
Pharmaceutical Benefits Scheme (PBS) from 1 October and follows a
positive recommendation from the Pharmaceutical Benefits Advisory
Committee (PBAC). A pathway to access for future Vertex CF medicine,
tezacaftor/ivacaftor, has been established as part of this process.

"We are delighted that lumacaftor/ivacaftor will be made available to
eligible CF patients in Australia," said Stuart Arbuckle, Executive Vice
President and Chief Commercial Officer at Vertex. "We would like to
recognize the PBAC for seeing the value of this medicine and acknowledge
the Department of Health and the Minister for Health for their active
engagement and willingness to finalize the agreement rapidly."

Australians with CF join thousands of patients worldwide who are already
receiving lumacaftor/ivacaftor. Beyond Australia, countries where
lumacaftor/ivacaftor is reimbursed include Austria, Denmark, Germany,
Ireland, Italy, the Netherlands, Sweden and the U.S.

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 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, 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. Arbuckle's statements 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 ORKAMBI 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)

View Comments and Join the Discussion!