PDUFA goal date extended by standard extension period of three months
to May 26, 2016
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 8, 2016--
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative
RNA-targeted therapeutics, today announced that the U.S. Food and Drug
Administration (FDA) will require additional time to complete its review
of the New Drug Application (NDA) for eteplirsen, for the treatment of
Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In a
notice received from the FDA, the Prescription Drug User Fee Act (PDUFA)
date for eteplirsen has been extended to May 26, 2016. The rescheduled
date for the Peripheral and Central Nervous System Advisory Committee
meeting has not yet been determined.
The FDA notified Sarepta that its January 8, 2016 submission of 4-year
clinical effectiveness data, which included additional six minute walk
test (6MWT) and loss of ambulation data compared to a historical
control, has been designated as a major amendment to the NDA. The FDA
stated that the PDUFA goal date has been extended by three months to
allow for a full review of the submission. As described in the Sarepta
Advisory Committee Briefing Document Addendum, the principal basis for
establishing the effectiveness of eteplirsen is a comparison of patients
in Study 201/202 to a historical control group.
“While our primary goal is to bring treatment to patients with Duchenne
as quickly as possible, we appreciate the efforts of the FDA to conduct
a complete review of all of the data supporting our NDA and we remain
committed to working closely with them throughout the remainder of the
regulatory process”, said Edward Kaye, Sarepta’s interim chief executive
officer and chief medical officer.
The FDA has previously granted eteplirsen Priority Review status, which
is designated for drugs that 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 percent 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.
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 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
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
rescheduled PDUFA goal date for an FDA decision on our NDA for
eteplirsen to allow for a full review by the FDA of the
additional 6MWT and loss of ambulation data submitted by the Company on
January 8, 2016 in the Sarepta Advisory Committee Briefing Document
Amendment which describes the Company’s principal basis for establishing
the effectiveness of eteplirsen is a comparison of patients in Study
201/202 to a historical control group; Sarepta’s primary goal of
bringing treatment to patients with Duchenne as quickly as possible and
its commitment to continue working closely with the FDA throughout the
remainder of the regulatory process.; and the potential market size 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 rescheduling or cancel the
Advisory Committee meeting or may further delay the PDUFA date for our
NDA for eteplirsen; any rescheduled Advisory Committee meeting may not
result in decisions or recommendations that result in the approval of
eteplirsen by the FDA; 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
additional research, clinical trials and commercialization plans of
eteplirsen and the development of 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, or
manufacturing issues; 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 September 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160208005533/en/
Source: Sarepta Therapeutics, Inc.
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan,
617-274-4052
iestepan@sarepta.com
or
W2O
Group
Ryan Flinn, 415-946-1059
Mobile: 510-207-7616
rflinn@w2ogroup.com
