-- Accelerated assessment of KALYDECO granted by the European
Medicines Agency; MAA submission planned in EU by the end of October --
-- Phase 3 registration studies of KALYDECO showed significant
improvements in lung function and other measures of disease among a
subset of people with CF --
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the
submission of a New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) for KALYDECO™ (VX-770, ivacaftor), a medicine in
development that targets the defective protein that causes cystic
fibrosis (CF). KALYDECO (kuh-LYE-deh-koh) was studied among people with
CF ages 6 and older who have at least one copy of the G551D mutation in
the cystic fibrosis transmembrane conductance regulator (CFTR)
gene. In the United States, approximately 4 percent of people with CF
are estimated to have at least one copy of the G551D mutation in the CFTR
gene.
Global Phase 3 pivotal studies of KALYDECO showed significant and
sustained improvements in lung function and other measures of disease in
people with CF who had at least one copy of the G551D mutation. The
majority of adverse events associated with KALYDECO were mild to
moderate in severity and non-serious. Fewer people in the KALYDECO
treatment groups than in the placebo groups discontinued treatment due
to adverse events. If approved, KALYDECO will be the first treatment to
target the underlying cause of CF.
The U.S. submission includes a request for Priority Review, which, if
granted, would shorten the FDA's anticipated review time from 10 to six
months. The FDA grants Priority Review status for several reasons,
including if the medicine is considered a major advance in treatment.
Vertex also plans to submit a marketing authorization application (MAA)
for KALYDECO with the European Medicines Agency (EMA) by the end of
October 2011. The EMA has accepted Vertex's request for accelerated
assessment, which is granted to new medicines of major public health
interest and shortens the review time from 210 days to 150 days
following the start of the review. Additionally, Vertex submitted
requests to the FDA and EMA to use the trade name KALYDECO (ivacaftor)
for VX-770.
"KALYDECO represents a completely new approach to the treatment of CF by
targeting the underlying cause of the disease," said Matthew Emmens,
Chairman, President and Chief Executive Officer of Vertex. "This is our
second new drug application in less than a year, which is a significant
achievement and underscores our commitment to developing new medicines
for people with serious diseases."
"The CF Foundation is thrilled that our collaboration with Vertex has
contributed to an important potential new medicine for the treatment of
some people with CF," said Robert J. Beall, Ph.D., President and CEO of
the CF Foundation. "The results of the KALYDECO studies have opened the
door to a new way of treating CF and we hope that this approach will
lead to the development of other targeted medicines for all people with
this disease."
CF is a life-threatening genetic disease that is caused by mutations in
the CFTR gene that result in defective or missing CFTR proteins.
The absence of functional CFTR proteins results in poor flow of salt and
water across cell membranes 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. Currently
available medicines have helped improve treatment and care for people
living with CF by treating the symptoms and some of the complications of
the disease.
Various mutations in the CFTR gene lead to CF. In some people,
CFTR proteins are present at the cell surface but do not function
properly. This dysfunction is known as a gating defect, the most common
of which is the G551D mutation. Approximately 4 percent of those with
CF, or about 1,200 people in the United States, are believed to have the
G551D mutation. KALYDECO is designed to keep the CFTR channels at the
cell surface open longer to improve the transport of chloride ions
across the cell membrane in people who have gating mutations. KALYDECO
has been studied in people with CF ages 6 and older who carry at least
one copy of the G551D mutation.
"These regulatory applications are a reflection Vertex's 13-year
research and development effort and the commitment of hundreds of
doctors, nurses, patients and their caregivers who participated in the
studies of KALYDECO," said Peter Mueller, Ph.D., Chief Scientific
Officer and Executive Vice President of Global Research and Development
at Vertex. "We look forward to working with U.S. and European regulatory
agencies to make KALYDECO available as quickly as possible."
Highlights of the KALYDECO Phase 3 Registration Program
These regulatory submissions are supported by results from two Phase 3
studies, STRIVE and ENVISION, in which people with CF who had at least
one copy of the G551D mutation and were treated with KALYDECO
experienced rapid, significant and sustained improvements across a
variety of disease measures, including lung function. These data support
the hypothesis that treating the underlying cause of CF may improve
outcomes for people with the disease. A Phase 2 study, DISCOVER, was
conducted in people who had two copies of the F508del mutation, the most
common CFTR mutation, and provided additional safety information
to support the regulatory applications for KALYDECO.
Overview of KALYDECO Discovery and Development Effort
KALYDECO was discovered as part of a collaboration with Cystic Fibrosis
Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and
development affiliate of the CF Foundation, to discover and develop
novel CFTR modulators.
Expanded Access Programs for KALYDECO
In recognition of the immediate needs of some people with CF, an
expanded access program for KALYDECO is currently open at participating
clinical trial sites in the United States. This program is designed to
provide KALYDECO to people ages 6 and older who have at least one copy
of the G551D mutation, are in critical medical need and may benefit from
treatment prior to potential FDA approval in the United States.
Vertex is working toward implementing additional expanded access
programs in other countries, with a goal of opening programs for
eligible patients by the end of 2011.
For more information, please call Vertex Medical Information at
1-877-634-VRTX (8789).
About Cystic Fibrosis
CF is a life-threatening genetic disease affecting approximately 30,000
people in the United States and 70,000 people worldwide. Today, the
median predicted age of survival for a person with CF is approximately
38 years. According to the 2010 Cystic Fibrosis Foundation Patient
Registry Annual Data Report, approximately 4 percent of the total CF
patient population in the United States have at least one copy of the
G551D mutation, 48 percent of the total CF patient population in the
United States have two copies of the F508del mutation and an additional
40 percent of the total CF patient population have one copy of the
F508del mutation.
Collaborative History with Cystic Fibrosis Foundation Therapeutics,
Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the Cystic Fibrosis Foundation. From 2000 through 2006,
Vertex and CFFT amended and expanded the collaboration four times to
support the accelerated discovery and development of KALYDECO and
VX-809. VX-809, known as a CFTR corrector, is designed to help the
protein reach the cell surface, while KALYDECO, known as a CFTR
potentiator, aims to help the protein function more normally once it
reaches the cell surface.
In April 2011, Vertex and CFFT further expanded the collaboration to
support development activities for VX-661, Vertex's second corrector to
enter clinical development, and the discovery and development of
next-generation correctors.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a
cure for cystic fibrosis. The Foundation funds more CF research than any
other organization and nearly every CF drug available today was made
possible because of Foundation support. Based in Bethesda, Md., the
Foundation also supports and accredits a national care center network
that has been recognized by the National Institutes of Health as a model
of care for a chronic disease. The CF Foundation is a donor-supported
nonprofit organization. For more information, visit www.cff.org.
About Vertex
Vertex creates new possibilities in medicine. Our team discovers,
develops and commercializes innovative therapies so people with serious
diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to
cure or significantly advance the treatment of hepatitis C, cystic
fibrosis, rheumatoid arthritis, epilepsy and other life-threatening
diseases.
Founded more than 20 years ago in Cambridge, MA, we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada.
Today, Vertex has more than 1,900 employees around the world, and Science
magazine named Vertex number one on its 2011 list of Top Employers in
the life sciences.
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 statements regarding (i) the plan to submit the registration
process for KALYDECO with the EMA by the end of October 2011; (ii) the
expectation that, if approved, KALYDECO will be the first treatment to
target the underlying cause of CF; (iii) the possibility that the FDA
review period for KALYDECO could be shortened from ten to six months;
(iv) Vertex's commitment to developing new medicines for people with
serious diseases; (v) the possibility that other targeted medicines
could be developed for all people with this disease; (vi) Vertex looking
forward to working with U.S. and European regulatory agencies to make
KALYDECO available as quickly as possible and (vii) the hypothesis that
treating the underlying cause of CF may improve outcomes for people with
CF. 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, that Vertex could experience
unforeseen delays in obtaining approval to market KALYDECO; that future
outcomes from clinical trials of KALYDECO may not be favorable; that
future scientific, clinical, competitive or other market factors may
adversely affect the potential for KALYDECO 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 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 Incorporated
Media:
Dawn
Kalmar/Megan Goulart/Zach Barber, 617-444-6992
mediainfo@vrtx.com
or
Investors:
Michael
Partridge, 617-444-6108
or
Lora Pike, 617-444-6755
or
Matthew
Osborne, 617-444-6057
Source: Vertex Pharmaceuticals Incorporated
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