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Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51
12/19/16 5:23 PM EST
“Around the world, there are many patients living with DMD who do not
have access to a medicine that treats the underlying cause of the
disease,” said
About Eteplirsen
Eteplirsen uses Sarepta’s proprietary
phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping
technology to skip exon 51 of the dystrophin gene. Eteplirsen is
designed to bind to exon 51 of dystrophin pre-mRNA, resulting in
exclusion of this exon during mRNA processing in patients with genetic
mutations that are amenable to exon 51 skipping. Exon skipping is
intended to allow for production of an internally truncated dystrophin
protein. Data from clinical studies of eteplirsen in a small number of
DMD patients have demonstrated a consistent safety and tolerability
profile. The pivotal trials were not designed to evaluate long-term
safety and a clinical benefit of eteplirsen has not been established.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare
degenerative neuromuscular disorder causing severe progressive muscle
loss and premature death. One of the most common fatal genetic
disorders, DMD affects approximately one in every 3,500-5,000 males
worldwide. A devastating and incurable muscle-wasting disease, DMD is
associated with specific errors in the gene that codes for dystrophin, a
protein that plays a key structural role in muscle fiber function.
Progressive muscle weakness in the lower limbs spreads to the arms, neck
and other areas. Eventually, increasing difficulty in breathing due to
respiratory muscle dysfunction requires ventilation support, and cardiac
dysfunction can lead to heart failure. The condition is universally
fatal, and death usually occurs before the age of 30.
About Sarepta Therapeutics
Sarepta Therapeutics is a
commercial-stage biopharmaceutical company focused on the discovery and
development of unique RNA-targeted therapeutics for the treatment of
rare neuromuscular diseases. The Company is primarily focused on rapidly
advancing the development of its potentially disease-modifying DMD drug
candidates. For more information, please visit us at www.sarepta.com.
Forward-Looking Statements
This press release contains
statements that are forward looking. Any statements contained in this
press release that are not statements of historical fact may be deemed
to be forward-looking statements. Words such as “believes,”
“anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,”
“looks,” “potential,” “possible” and similar expressions are intended to
identify forward-looking statements. These forward-looking statements
include statements regarding Sarepta seeking conditional approval of
eteplirsen in the EU, the validation starting the formal review process
by the EMA’s CHMP, the standard 210 day review period, and that the
validation of the MAA is the next step toward Sarepta’s goal of
providing potential therapies to more patients with Duchenne in
These forward-looking statements involve risks and uncertainties, many
of which are beyond Sarepta’s control. Actual results could materially
differ from those stated or implied by these forward-looking statements
as a result of such risks and uncertainties. Known risk factors include
the following: the EMA may delay its decision beyond the standard MAA
review or may determine that our MAA submission for eteplirsen does not
qualify for approval; the results of our clinical trials and additional
information and data we collect for the eteplirsen and our other product
candidates may not be consistent with prior data or results, may not be
positive and/or may not support the safety and efficacy of eteplirsen,
our other product candidates and/or Sarepta’s anti-sense based
technology platform; the estimates regarding the market size for
eteplirsen or any of our product candidates may not be correct; we may
not be able to achieve or there may be delays in our projected
regulatory and development timelines for eteplirsen including satisfying
EMA requests related to our eteplirsen MAA filing, our clinical studies,
our planned meetings and discussions with regulatory authorities, our
plans for commercializing eteplirsen in the E.U., the development of our
other product candidates for various reasons including possible
limitations of Company financial and other resources and regulatory,
court or agency decisions, such as decisions with respect to patents
that cover our product candidates; factors that would negatively impact
our manufacturing efforts; and those risks identified under the heading
“Risk Factors” in Sarepta’s 2015 Annual Report on Form 10-K or most
recent Quarterly Report on Form 10-Q filed with the
Any of the foregoing risks could materially and adversely affect
Sarepta's business, results of operations and the trading price of
Sarepta's common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review the Company's
filings with the
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this release are forward–looking statements that involve risks and
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View source version on businesswire.com: http://www.businesswire.com/news/home/20161219006266/en/
Source:
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan,
617-274-4052
iestepan@sarepta.com
or
W2O
Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission.