Vertex Announces Acceptance of its Applications for Review of the Tezacaftor/Ivacaftor Combination Treatment in People with C...
August 24 2017 - 11:30AM
Business Wire
-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-
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)
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Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6470orMedia:North America:Megan Goulart, +
1-617-341-6992mediainfo@vrtx.comorEurope & Australia:Rebecca
Hunt, +44 7718 962690
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