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Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for Eteplirsen After Further Review of Data on Dystrophin

Sarepta Therapeutics Announces FDA Will Consider Accelerated Approval for 
Eteplirsen After Further Review of Data on Dystrophin and
Clinical Outcomes 
Eteplirsen Manufacturing and Clinical Activities Continue as Planned 
CAMBRIDGE, MA -- (Marketwired) -- 04/15/13 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT) today provided an update on its discussions with
the U.S. Food and Drug Administration (FDA) regarding a potential
application for accelerated approval of eteplirsen for the treatment
of Duchenne muscular dystrophy (DMD). The FDA has requested that
Sarepta provide additional information from the existing eteplirsen
dataset to inform a decision on the acceptability of this dataset for
a New Drug Application (NDA) filing under the Subpart H Accelerated
Approval regulatory pathway. This feedback was provided in meeting
minutes from Sarepta's End-of-Phase II meeting with the FDA's
Division of Neurology Products that occurred last month.  
Specifically, Sarepta received feedback on both the acceptability of
dystrophin as a surrogate endpoint that would reasonably predict
clinical benefit in DMD patients and the acceptability of the
eteplirsen safety database for a Subpart H Accelerated Approval
filing. 
"We are encouraged by our interactions with the FDA and their
Division of Neurology Products and we view their request for more
data as a reflection of the thorough and comprehensive approach that
the Agency takes in evaluating a new surrogate marker," said Chris
Garabedian, president and chief executive officer of Sarepta
Therapeutics. "We are confident that the method in which we've
collected dystrophin, the degree and consistency of the dystrophin
levels, and the supporting clinical data will provide the Agency the
information it needs to determine if dystrophin is a feasible
surrogate marker that is reasonably likely to predict clinical
benefit." 
At the End-of-Phase II Clinical Meeting in March, there was a
productive discussion on the suitability of dystrophin as a surrogate
marker, including a presentation by Sarepta detailing the
methodologies used to analyze dystrophin in the studies and
supportive data suggesting that the dystrophin produced is
functional. As follow up to the discussion, and as reflected in the
meeting minutes from the Division of Neurology Products, the Agency
requested two written summaries: "a coherent and comprehensive
summary to support dystrophin as a surrogate" and "a detailed
discussion of all clinical outcomes in the eteplirsen study."  
Furthermore, the meeting minutes contained the following statement:  
"The Agency stated that they had not made a final decision regarding
acceptability of the proposed Subpart H (Accelerated Approval) NDA
filing, and that the Agency would consider the additional data
submitted by the sponsor before making a final decision." 
Sarepta also discussed the eteplirsen and phosphorodiamidate
morpholino oligomer (PMO) safety database at the End-of-Phase II
meeting and asked if the 38 patient eteplirsen safety database was
sufficient for a Subpart H Accelerated Approval filing. While Sarepta
will continue to collect long-term safety from the ongoing eteplirsen
extension study for a potential submission, the meeting minutes
stated that: "In the event (the Agency) agrees to file the Subpart H
NDA submission, additional safety data to support approval could come
from the first few months of the... pivotal confirmatory study."
Sarepta still intends to begin dosing patients in a pivotal
confirmatory study in the first quarter of 2014. 
Sarepta is preparing to submit the dystrophin and clinical outcomes
summaries and will be requesting a follow-up meeting with the FDA to
discuss these later this quarter. As a result, the End-of-Phase II
CMC meeting with the FDA is now expected to occur in the third
quarter, however all eteplirsen manufacturing and clinical
development activities continue as planned and are not anticipated to
delay the potential timeline of an Accelerated Approval NDA
submission. 
Conference Call Information 
Sarepta will hold a conference call to discuss this update today at
5:00 p.m. EDT. The conference call may be accessed by dialing
800.446.2782 for domestic callers and 847.413.3235 for international
callers. The passcode for the call is 34696256. Please specify to the
operator that you would like to join the "Sarepta Therapeutics
Corporate Conference Call." The conference call will be webcast live
under the events section of Sarepta's website at
www.sareptatherapeutics.com and will be archived there following the
call for 90 days. Please connect to Sarepta's website several minutes
prior to the start of the broadcast to ensure adequate time for any
software download that may be necessary. An audio replay will be
available through May 9, 2013 by calling 888.843.7419 or 630.652.3042
and entering access code 34696256. 
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 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 eventually 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 Eteplirsen 
Eteplirsen is Sarepta's lead drug candidate that is designed to
address the underlying cause of DMD by enabling the production of a
functional dystrophin protein. Eteplirsen uses Sarepta's
phosphorodiamidate morpholino oligomer (PMO)-based chemistry and
exon-skipping technology to skip exon 51 of the dystrophin gene
enabling the repair of specific genetic mutations that affect
approximately 13 percent 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 improve, stabilize or significantly slow the disease process and
prolong and improve the quality of life for patients with DMD. 
About Sarepta Therapeutics 
Sarepta Therapeutics is focused on developing first-in-class
RNA-based therapeutics to improve and save the lives of people
affected by serious and life-threatening rare and infectious
diseases. The Company's diverse pipeline includes its lead program
eteplirsen, for Duchenne muscular dystrophy, as well as potential
treatments for some of the world's most lethal infectious diseases.
Sarepta aims to build a leading, independent biotech company
dedicated to translating its RNA-based science into transformational
therapeutics for patients who face significant unmet medical needs.
For more information, please visit us at www.sareptatherapeutics.com. 
About the Accelerated Approval Regulatory Pathway (21 CFR 314 Subpart
H) 
To speed the development and availability of new medicines for
serious and life-threatening diseases, the FDA has the authority to
grant accelerated approval based on evidence that an investigational
new drug provides a meaningful therapeutic benefit over existing
treatments. Under this authority, marketing approval may be granted
on the basis of adequate and well-controlled clinical trials that
show the drug has an effect on a surrogate endpoint that is
reasonably likely to predict clinical benefit or on the basis of a
clinical endpoint other than survival or irreversible morbidity. If
granted, the FDA requires post-marketing studies to verify the
clinical benefit of the approved medicine. For more information,
visit the FDA web site at:
http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=31
4&showFR=1&subpartNode=21:5.0.1.1.4.8. 
Forward Looking Statement 
This press release contains forward-looking statements. These
forward-looking statements generally can be identified by use of
words such as "believes or belief," "anticipates," "plans,"
"expects," "will," "intends," "potential," "possible," "advance" and
similar expressions. These forward-looking statements include
statements about the development of eteplirsen and its efficacy,
safety, potency and utility in the treatment of DMD; the potential
for the creation of novel dystrophin to lead to significant clinical
benefit or its suitability as a valid surrogate marker; the amount
and type of dystrophin, safety and other information necessary for
the Agency to make regulatory determinations; the impact of
manufacturing and development activities on NDA submission timelines;
and the potential use of the accelerated approval pathway for
eteplirsen. 
Each forward-looking statement contained in this press release is
subject to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied by such statement.
Applicable risks and uncertainties include, among others: subsequent
clinical trials may fail to demonstrate the safety and efficacy of
eteplirsen or replicate results; treatment of patients with DMD using
eteplirsen over a longer duration may not lead to significant
clinical benefit; any of Sarepta's drug candidates, including
eteplirsen, may fail in development, may not qualify for filing under
Subpart H accelerated approval or receive required regulatory
approvals at all, or may not become commercially viable due to
delays, decisions by regulatory authorities, or other reasons; and
those identified under the heading "Risk Factors" in Sarepta's Annual
Report on Form 10-K for the full year ended December 31, 2012, and
filed with the Securities and Exchange Commission.  
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 Securities and Exchange Commission. 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. 
"Safe Harbor" Statement under the Private Securities Litigation
Reform Act of 1995: The statements that are not historical facts
contained in this release are forward-looking statements that involve
risks and uncertainties, including, but not limited to, the results
of research and development efforts, the results of preclinical and
clinical testing, the effect of regulation by the FDA and other
agencies, the impact of competitive products, product development,
commercialization and technological difficulties, and other risks
detailed in the company's Securities and Exchange Commission filings. 
Sarepta Investor Contact: 
Erin Cox 
857.242.3714 
ecox@sareptatherapeutics.com 
Sarepta Media Contact:
Jim Baker
857.242.3710
jbaker@sareptatherapeutics.com 
 
 
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