Food & Drug Administration Advisory Panel Voted 12 to 1 to Recommend Approval of ORKAMBI™ (lumacaftor/ivacaftor) to Treat P...
May 12 2015 - 4:15PM
Business Wire
-FDA decision expected by July 5, 2015 PDUFA
date-
-Approximately 8,500 people with cystic
fibrosis in the U.S. have two copies of the F508del mutation and
are ages 12 and older-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration’s (FDA)
Pulmonary-Allergy Drugs Advisory Committee (PADAC) voted 12 to 1 to
recommend that the FDA approve ORKAMBITM (lumacaftor/ivacaftor) for
use in people with cystic fibrosis (CF) ages 12 and older who have
two copies of the F508del mutation in the CFTR gene. Advisory
committees provide the FDA with independent scientific and medical
advice on safety, effectiveness and appropriate use of potential
new medicines. The FDA is expected to make a decision on the
approval of ORKAMBI by July 5, 2015 under the Prescription Drug
User Fee Act (PDUFA). The FDA is not bound by the committee's
recommendation but often follows its advice. If approved, ORKAMBI
will be the first and only medicine to treat the underlying cause
of CF for eligible people with CF ages 12 and older with two copies
of the F508del mutation in the CFTR gene. People with two copies of
the F508del mutation represent the largest group of people with CF.
There are approximately 8,500 people ages 12 and older with two
copies of the F508del mutation in the U.S.
“Today’s positive recommendation brings the cystic fibrosis
community one step closer to potential approval of the first
medicine to treat the underlying cause of this disease for many
more people,” said Jeffrey Chodakewitz, M.D., Executive Vice
President and Chief Medical Officer at Vertex. “We look forward to
continuing to work with the FDA and other regulatory agencies
throughout the world to make ORKAMBI available to eligible patients
as soon as possible.”
Cystic fibrosis is a rare genetic disease that is caused by
defective or missing CFTR proteins resulting from mutations in
the CFTR gene. The defective or missing proteins result
in poor flow of salt and water into and out of the cell in a number
of organs, including the lungs. 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.
ORKAMBI is a combination of lumacaftor, which is designed to
increase the amount of functional protein at the cell surface by
addressing the processing and trafficking defect of the protein,
and ivacaftor, which is designed to enhance the function of the
CFTR protein once it reaches the cell surface. ORKAMBI is an oral
medicine that, if approved, would be taken as fully co-formulated
tablets that contain both lumacaftor and ivacaftor.
About Cystic Fibrosis
CF is a rare, life-threatening genetic disease affecting
approximately 75,000 people in North
America, Europe and Australia. Children must inherit
two defective CFTR genes — one from each parent — to have
CF. There are more than 1,900 known mutations in
the CFTR gene. Some of these mutations, which can be
determined by a genetic, or genotyping, test, lead to CF by
creating non-working or too few CFTR proteins at the cell surface.
The defective or missing CFTR protein results in poor flow of salt
and water into and out of the cell in a number of organs, including
the lungs. This leads to the buildup of abnormally thick, sticky
mucus that can cause chronic lung infections and progressive lung
damage, eventually leading to death.
Today, the median predicted age of survival for a person with CF
is between 34 and 47 years, but the median age of death remains in
the mid-20s.
Collaborative History with Cystic Fibrosis Foundation
Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a
collaboration with Cystic Fibrosis Foundation Therapeutics (CFFT),
the nonprofit drug discovery and development affiliate of
the Cystic Fibrosis Foundation. This collaboration was
expanded to support the accelerated discovery and development of
Vertex's CFTR modulators.
About Vertex
Vertex is a global biotechnology company that aims to discover,
develop and commercialize innovative medicines so people with
serious diseases can lead better lives. In addition to our clinical
development programs focused on cystic fibrosis, Vertex has more
than a dozen ongoing research programs aimed at other serious and
life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has
research and development sites and commercial offices in the
United States, Europe, Canada and Australia. For
five years in a row, Science magazine has named Vertex
one of its Top Employers in the life sciences. For additional
information and the latest updates from the company, please
visit www.vrtx.com.
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, statements regarding the potential
approval of ORKAMBI as a treatment for patients with CF twelve
years or older who have two copies of the F508del mutation in their
CFTR gene. 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, ORKAMBI for patients with CF twelve years or older who have
two copies of the F508del mutation in their CFTR gene 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)
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
617-341-6108orKelly Lewis, 617-961-7530orEric Rojas,
617-961-7205orMedia:Zach Barber, mobile: 617-767-9533,
office: 617-341-6992mediainfo@vrtx.com
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