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FDA Grants Priority Review Status
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PDUFA Date is February 26, 2016
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative
RNA-targeted therapeutics, today announced that the U.S. Food and Drug
Administration (FDA) has filed the New Drug Application (NDA) for
eteplirsen for the treatment of Duchenne muscular dystrophy (DMD)
amenable to exon 51 skipping. Approximately 13% of people with Duchenne
muscular dystrophy are estimated to have a mutation addressable by
Eteplirsen/exon 51 skipping.
The FDA has completed its filing review and has determined that our
application is sufficiently complete to permit a substantive review. The
Prescription Drug User Fee Act (PDUFA) action date for a decision on the
application is February 26, 2015. The FDA has granted eteplirsen
Priority Review status, which is designated to drugs that offer benefit
over existing therapies, or provide a treatment where no adequate
therapy exists.
“We are pleased with the FDA’s acceptance of our NDA for eteplirsen, as
it represents an important milestone, not only for Sarepta, but for the
Duchenne community. We look forward to continuing to work closely with
the FDA during the regulatory review process,” said Edward M. Kaye,
interim chief executive officer and chief medical officer. “We believe
eteplirsen has the potential to make a meaningful impact on the lives of
patients amenable to skipping exon 51 and we aim to build on our
experience with eteplirsen to work with the FDA to inform and
potentially expedite the clinical and regulatory pathway for the follow
on exons, with the goal of reaching as many patients amenable to exon
skipping 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 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 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 the potential DMD
population that is amenable to exon 51 skipping, the potential market
size for eteplirsen, the PDUFA date for the eteplirsen NDA, Sarepta’s
plans to continue working closely with the FDA in the regulatory review
process, the potential meaningful impact of eteplirsen on the lives of
patients amenable to exon 51 skipping, Sarepta’s plans to build on its
experience with eteplirsen and work with the FDA to inform and
potentially expedite the clinical and regulatory pathway for follow-on
exons and Sarepta’s goal to reach as many patients with DMD amenable to
exon skipping as possible.
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 estimates regarding the market size
for eteplirsen or any of our product candidates may not be correct; the
FDA may determine that our NDA submission for eteplirsen does not
qualify for approval; an advisory committee, if any is convened, may not
provide a positive recommendation to the FDA for eteplirsen; 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; there
may be delays in our projected regulatory and development timelines
relating to our eteplirsen NDA, clinical studies, our planned meetings
and discussions with the FDA, and plans for commercializing eteplirsen
and 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 by the United States Patent
and Trademark Office with respect to patents that cover our product
candidates; scale-up of manufacturing may not be successful and any or
all of the Company’s drug candidates may fail in development or may not
receive required regulatory approvals for commercialization (including
potentially under an accelerated pathway); and those risks identified
under the heading “Risk Factors” in Sarepta’s 2014 Annual Report on Form
10-K or most recent Quarterly Report on Form 10-Q 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 the
Company’s business, results of operations and the trading price of
Sarepta’s common stock. You should not place undue reliance on
forward-looking statements. Sarepta does not undertake any obligation to
publicly update its forward-looking statements based on events or
circumstances after the date hereof, except to the extent required by
applicable law or SEC rules.
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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