-FDA grants Priority Review of the application and sets action date
of February 28, 2018-
-Applications supported by positive results from two global Phase 3
studies in people with CF ages 12 and older who have two copies of the
F508del mutation or one F508del mutation and one residual function
mutation that is responsive to tezacaftor/ivacaftor-
BOSTON--(BUSINESS WIRE)--
Vertex
Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the
acceptance of its applications for the use of the tezacaftor/ivacaftor
combination treatment in people with cystic fibrosis (CF) ages 12 and
older who have two copies of the F508del mutation or one F508del
mutation and one residual function mutation that is responsive to
tezacaftor/ivacaftor by the U.S. Food and Drug Administration (FDA) and
the European Medicines Agency (EMA). In the United States, the FDA has
granted Priority Review of the New Drug Application (NDA) and set an
action date of February 28, 2018. The submissions are supported by
positive results from two global Phase
3 studies.
"If approved, the tezacaftor/ivacaftor combination treatment would
become Vertex's third medicine to treat the underlying cause of cystic
fibrosis, offering an important new treatment option for a large group
of patients with this rare and life-shortening disease," said Jeffrey
Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at
Vertex. "We look forward to working with the agencies to facilitate a
rapid review of these applications."
In the United States, the tezacaftor/ivacaftor combination treatment was
granted Priority Review designation, which shortens the FDA's
anticipated review time from approximately 12 months to eight months.
The combination treatment also received Breakthrough Therapy
Designation. Breakthrough Therapy Designation is intended to expedite
the development and review of drugs for serious or life-threatening
conditions. In the European Union, the EMA has validated the Marketing
Authorization Application (MAA), which confirms that the submission is
complete and initiates the centralized review process of approximately
210 days for the Committee for Medicinal Products for Human Use (CHMP)
to give an Opinion. The CHMP Opinion is then reviewed by the European
Commission, which has the authority to approve medicines for the
European Union and generally issues a final decision within two to three
months. If approved, Vertex would then begin the country-by-country
reimbursement approval process.
About Cystic Fibrosis
Cystic Fibrosis (CF) 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 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.
In people with the F508del mutation, the CFTR protein
is not processed, or folded, normally within the cell and generally does
not reach the cell surface. Tezacaftor is designed to address the
processing defect of F508del-CFTR to enable it to reach the cell surface
where ivacaftor can further enhance the protein's function.
In North America, Europe and Australia, there are more than 22,000
people ages 12 and older who have two copies of the F508del
mutation, and there are more than 1,500 people ages 12 and older who
have at least one residual function mutation that is responsive to
tezacaftor/ivacaftor in vitro or in the clinic.
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 seven years in a row. For
additional information and the latest updates from the company, please
visit www.vrtx.com.
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) and tezacaftor 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, including, without
limitation, Dr. Chodakewitz's statements in the second paragraph of the
press release, the FDA's target action date and information regarding
the review process in the United States and European Union. 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 regulatory authorities may not approve, or approve on
a timely basis, the NDA or the MAA, that data from the company's
development programs may not support registration or further development
of its compounds due to safety, efficacy or other reasons, and other
risks listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and available
through the company's website at www.vrtx.com.
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
View source version on businesswire.com: http://www.businesswire.com/news/home/20170824005862/en/
Vertex Pharmaceuticals Incorporated
Investors:
Michael
Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach
Barber, 617-341-6470
or
Media:
North America:
Megan
Goulart, + 1-617-341-6992
mediainfo@vrtx.com
or
Europe & Australia:
Rebecca
Hunt, +44 7718 962690
Source: Vertex Pharmaceuticals Incorporated
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