Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date
May 25 2016 - 7:00AM
Business Wire
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of
innovative RNA-targeted therapeutics, today announced that the U.S.
Food and Drug Administration (FDA) has notified the Company that
they are continuing their review and internal discussions related
to our pending NDA for eteplirsen and will not be able to complete
their work by the Prescription Drug User Fee Act (PDUFA) goal date
of May 26, 2016. The FDA has communicated that they will continue
to work past the PDUFA goal date and strive to complete their work
in as timely a manner as possible.
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.
About Eteplirsen
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 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.
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 FDA not completing its
review of the eteplirsen NDA by the PDUFA date of May 26, 2016,
additional review and internal FDA discussions relating to the NDA,
and the timing for the FDA completing its work and reaching a
decision with respect to the Company’s eteplirsen NDA.
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 its
decision on the eteplirsen NDA or may not provide marketing
approval for eteplirsen; 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 our
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 factors outside of the Company’s control, 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 Report on Form 10-Q
for the quarter ended March 31, 2016 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20160525005442/en/
Sarepta Therapeutics, Inc.Media and Investors:Ian Estepan,
617-274-4052iestepan@sarepta.comorW2O GroupRyan Flinn,
415-946-1059Mobile: 510-207-7616rflinn@w2ogroup.com
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