Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative
RNA-targeted therapeutics, today announced the Peripheral and Central
Nervous System Drugs Advisory Committee of the U.S. Food and Drug
Administration (FDA) is tentatively scheduled to review Sarepta’s New
Drug Application (NDA) for eteplirsen, for the treatment of Duchenne
muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22,
2016. There has been no formal communication to the company or Federal
Register notice of a confirmed advisory committee meeting date.
It is estimated that DMD affects approximately one in every 3,500 boys
born worldwide, with 13% of people with the disease having mutations
addressable by eteplirsen/exon 51 skipping.
“We have been verbally informed by the FDA that we have a tentative date
of January 22, 2016 for our advisory committee review of eteplirsen and
our PDUFA date of February 26, 2016, remains unchanged.” said Edward
Kaye, M.D., Sarepta’s interim chief executive officer and chief medical
officer. “We look forward to discussing the efficacy and safety data
included in our NDA submission for eteplirsen with the committee, with
the ultimate goal of bringing treatment to more patients with Duchenne.”
About Sarepta Therapeutics
Sarepta Therapeutics is a biopharmaceutical company focused on the
discovery and development of unique RNA-targeted therapeutics for the
treatment of rare, infectious and other diseases. The Company is
primarily focused on rapidly advancing the development of its
potentially disease-modifying DMD drug candidates, including its lead
DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is
also developing therapeutics for the treatment of infectious diseases,
such as drug-resistant bacteria and other rare human diseases. For more
information, please visit us at www.sarepta.com.
About Eteplirsen
Eteplirsen is Sarepta's lead drug candidate and is designed to address
the underlying cause of DMD by enabling the production of a functional
dystrophin protein. Data from clinical studies of eteplirsen in DMD
patients have demonstrated a broadly favorable safety and tolerability
profile and restoration of dystrophin protein expression.
Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer
(PMO)-based chemistry and proprietary exon-skipping technology to skip
exon 51 of the dystrophin gene enabling the repair of specific genetic
mutations that affect approximately 13% of the total DMD population. By
skipping exon 51, eteplirsen may restore the gene's ability to make a
shorter, but still functional, form of dystrophin from messenger RNA, or
mRNA. Promoting the synthesis of a truncated dystrophin protein is
intended to stabilize or significantly slow the disease process and
prolong and improve the quality of life for patients with DMD.
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 boys born 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.
Forward-Looking Statements
This press release contains "forward-looking statements" within the
meaning of the safe harbor provisions of the U.S. Private Securities
Litigation Reform Act of 1995. 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," "intends," "potential," "possible" and
similar expressions are intended to identify forward-looking statements.
These forward-looking statements include statements regarding the
potential date for a meeting of an FDA advisory committee to review the
NDA for eteplirsen and the applicable PDUFA date, the potential market
for eteplirsen, Sarepta’s planned discussions on efficacy and safety
data included in the NDA submission with the Committee and Sarepta’s
ultimate goal of bringing treatment to more patients with Duchenne.
Forward-looking statements also include those regarding Sarepta’s future
business developments and actions and the timing of the same.
These forward-looking statements involve risks and uncertainties,
many of which are beyond Sarepta's control. Known risk factors include,
among others: the FDA may further delay or cancel the tentative advisory
committee meeting, the results of our ongoing research and development
efforts and clinical trials for eteplirsen and our other product
candidates may not be positive or consistent with prior results or
demonstrate a safe treatment benefit, there may be delays in Sarepta's
projected regulatory and development timelines relating to the
eteplirsen NDA and plans for commercializing eteplirsen and developing
Sarepta's other product candidates for various reasons including
possible limitations of Sarepta's financial and other resources; Sarepta
may not be able to successfully complete its planned commercialization
of eteplirsen or continue developing its product candidates as planned
for a variety of reasons including due to regulatory, court or agency
decisions, such as decisions by the USPTO with respect to patents that
cover Sarepta's product candidates, scale-up of manufacturing may not be
successful, and any or all of Sarepta's product candidates may
fail in development or may not receive required regulatory approvals for
commercialization; and those risks identified under the heading “Risk
Factors” in Sarepta's 2014 Annual Report on Form 10-K or and most recent
Quarterly Report on Form 10-Q for the quarter ended June 30, 2015 filed
with the Securities and Exchange Commission (SEC) as well as other SEC
filings made by the Company which you are encouraged to review.
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 SEC. We caution investors not to place considerable
reliance on the forward-looking statements contained in this press
release. Sarepta does not undertake any obligation to publicly update
its forward-looking statements based on events or circumstances after
the date hereof.
Internet Posting of Information
We routinely post information that may be important to investors in
the 'For Investors' section of our website at www.sarepta.com.
We encourage investors and potential investors to consult our website
regularly for important information about us.
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