-- Six-month review date of April 18, 2012 set by FDA --
-- European Medicines Agency accepts KALYDECO
regulatory submission for accelerated assessment --
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that
the U.S. Food and Drug Administration (FDA) has accepted the New Drug
Application (NDA) for KALYDECOTM (ivacaftor) and granted the
company's request for six-month Priority Review. KALYDECO targets the
defective protein that causes cystic fibrosis (CF) in a subset of people
with the disease. If approved, KALYDECO will be the first treatment to
target the underlying cause of CF.
The FDA grants Priority Review to medicines that offer major advances in
treatment or provide a treatment where no adequate therapy exists. A
target review date of April 18, 2012 is set under the Prescription Drug
User Fee Act (PDUFA) for the FDA's approval decision, which is four
months earlier than the standard review time of 10 months.
In addition, Vertex announced today that its marketing authorization
application (MAA) for KALYDECO has been validated by the European
Medicines Agency (EMA). Validation indicates that the application is
complete and starts the regulatory review process by the Committee for
Medicinal Products for Human Use (CHMP). Earlier this year, the EMA
accepted Vertex's request for accelerated assessment, which is granted
to new medicines of major public health interest and shortens the EMA's
"If approved, KALYDECO will be the first treatment to target the
underlying cause of CF," said Peter Mueller, Ph.D., Chief Scientific
Officer and Executive Vice President of Global Research and Development
at Vertex. "The commitments by the FDA and the EMA to expedite their
reviews of our applications underscore the significant potential of
KALYDECO to help people living with cystic fibrosis."
CF is caused by defective or missing cystic fibrosis transmembrane
conductance regulator (CFTR) proteins resulting from mutations in the CFTR
gene. 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.
In people with CF who have a gating defect, CFTR proteins are present at
the cell surface but do not function properly. The most common gating
defect is caused by the G551D mutation. Approximately 4 percent of those
with CF, or about 1,200 people in the United States and 1,000 people in
Europe, are believed to have this 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. The U.S. application seeks approval for KALYDECO in
people with the G551D mutation. The application submitted in Europe
includes a request for all gating mutations.
The 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. 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.
These data showed that treating the underlying cause of CF may improve
outcomes for people with the disease.
KALYDECO (ivacaftor, VX-770) is Vertex's lead medicine in development
for the treatment of people with cystic fibrosis. Known as a CFTR
potentiator, this oral medicine in development aims to help CFTR protein
function more normally once it reaches the cell surface, which is
believed to help hydrate and clear mucus from the airways. Vertex
retains worldwide rights to develop and commercialize KALYDECO
(kuh-LYE-deh-koh). The brand name KALYDECO has been approved by the EMA
and provisionally approved by the FDA, but KALYDECO has not been granted
marketing authorization or approval from any regulatory authority.
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. The most common form of CF is caused by the F508del
mutation, which is present in nearly 90 percent of people with the
Collaborative History with Cystic Fibrosis Foundation Therapeutics,
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. This collaboration was
expanded to support the accelerated discovery and development of
Vertex's CFTR modulators.
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. Many of the CF treatments available today benefited
from 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.
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
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, including
statements regarding (i) the FDA's target review date for the KALYDECO
NDA, and (ii) the commitments by the FDA and EMA to expedite their
reviews of our applications underscoring the significant potential of
KALYDECO to help people living with cystic fibrosis. 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 scientific, clinical,
competitive or other market factors may adversely affect the potential
for therapy with 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.
Vertex Pharmaceuticals Incorporated
Lora Pike, 617-444-6755
Source: Vertex Pharmaceuticals Incorporated
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