--Advisory Committee Meeting Rescheduled for April 25, 2016--
--PDUFA date for eteplirsen is May 26, 2016--
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 14, 2016--
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative
RNA-targeted therapeutics, today announced that the Peripheral and
Central Nervous System (PCNS) Drugs Advisory Committee of the U.S. Food
and Drug Administration (FDA) will review Sarepta’s New Drug Application
(NDA) for eteplirsen on April 25, 2016. The Prescription Drug User Fee
Act (PDUFA) action date for completion of FDA review of the eteplirsen
is May 26, 2016.
The FDA has granted eteplirsen Priority Review status, which is
designated for drugs which provide a treatment where no adequate therapy
exists. The FDA also granted Rare Pediatric Disease Designation to
eteplirsen, as well Orphan Drug Designation and Fast Track Status.
It is estimated that Duchenne muscular dystrophy affects approximately
one in every 3,500 – 5,000 boys born worldwide, with 13% of people with
the disease having mutations addressable by eteplirsen/exon 51 skipping.
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 rare, infectious and
other diseases. For more information, please visit us at www.sarepta.com.
Eteplirsen is designed to address the underlying cause of DMD by
restoring the dystrophin messenger RNA (mRNA) reading frame, thus
enabling the production of a shorter, functional form of the dystrophin
protein. Eteplirsen uses Sarepta’s proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to skip
exon 51 of the dystrophin gene. Approximately 13 percent of the DMD
population is amenable to exon 51 skipping. Data from clinical studies
of eteplirsen in DMD patients have demonstrated a consistent safety and
tolerability profile and have also shown measurable dystrophin protein
expression. Promoting the synthesis of a shorter dystrophin protein is
intended to slow the decline of ambulation and mobility seen in DMD
patients. There currently is no approved treatment in the United States
for DMD and eteplirsen has not been approved by the FDA or any
regulatory authority for the treatment of 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
3,500-5,000 boys 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
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
Advisory Committee date to review the NDA for eteplirsen and the
applicable PDUFA date and the potential market for eteplirsen.
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 Advisory Committee
meeting; the Advisory Committee may not recommend eteplirsen for
approval and even if they do, the FDA may not provide eteplirsen with
marketing approval by the applicable PDUFA date or at all; we may not be
able to comply with all FDA requests, including with respect to our
eteplirsen NDA submission and the addendums we have submitted to the FDA
or with respect to our ongoing or planned clinical trials, in a timely
manner or at all; we may not be able to complete clinical trials
required by the FDA for approval of our products or any submissions made
in connection with our pipeline of product candidates; the results of
our ongoing research and development efforts and clinical trials for our
product candidates including eteplirsen and technologies may not be
positive or consistent with prior results or demonstrate a safe
treatment benefit or support an NDA filing, positive advisory committee
recommendation or marketing approval by the FDA or other regulatory
authority; we may not be able to execute on our business plans including
meeting our expected or planned regulatory milestones and timelines,
clinical development plans and bringing our product candidates to
market, including the planned commercialization of eteplirsen, for
various reasons including possible limitations of Company financial and
other resources, manufacturing limitations that may not be anticipated
or resolved for in a timely manner or at all, and regulatory, court or
agency decisions, such as decisions by the United States Patent and
Trademark Office with respect to patents that cover our product
candidates; and those risks identified under the heading “Risk Factors”
in Sarepta’s most recent Annual Report on Form 10-K for the year ended
December 31, 2015 or Quarterly Reports on Form 10-Q filed with
the Securities and Exchange Commission (SEC) as well as other
SEC filings made by Sarepta 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160314005593/en/
Source: Sarepta Therapeutics, Inc.
Media and Investors:
Sarepta Therapeutics, Inc.
Ryan Flinn, 415-946-1059