Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for
Eteplirsen for the Treatment of Duchenne Muscular
Dystrophy in First Half of 2014
CAMBRIDGE, MA -- (Marketwired) -- 07/24/13 -- Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced it plans to submit a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA) in
the first half of 2014 for the approval of eteplirsen for the
treatment of Duchenne muscular dystrophy (DMD). Eteplirsen is
Sarepta's lead exon-skipping compound in development for the
treatment of patients with DMD who have a genotype amenable to
skipping of exon 51.
The decision to submit an NDA for eteplirsen in 2014 is based on
productive interactions with the FDA in a meeting that occurred this
week. That meeting was a follow-up to the FDA's review of two
recently submitted summary documents that included data on dystrophin
and clinical outcomes from the existing eteplirsen studies. The FDA
stated in pre-meeting comments that the Agency is "open to
considering an NDA based on these data for filing." The Agency,
however, requested additional information related to the methodology
and verification of dystrophin quantification. Sarepta believes the
requests from the Agency can be addressed and incorporated into an
NDA submission in the first half of 2014.
"We are encouraged by the feedback from the FDA and believe that data
from our ongoing clinical study merits review by the Agency and will
be sufficient for an NDA filing," said Chris Garabedian, president
and chief executive officer of Sarepta Therapeutics. "We plan to work
closely with the FDA to prepare an NDA submission in the first half
of 2014 as we continue to prepare for our confirmatory study and our
manufacturing scale up."
The Agency would not commit to declaring dystrophin an acceptable
surrogate endpoint under the CFR 314 Subpart H Accelerated Approval
pathway prior to an NDA filing and commented that a decision by the
Agency to file "the NDA would not indicate that we have accepted
dystrophin expression as a biomarker reasonably likely to predict
clinical benefit. A filing would only indicate that the question
merits review, and that we deem the data to be reviewable."
Sarepta anticipates submitting an NDA for eteplirsen in the first
half of 2014; however, the exact timing of the submission will be
dependent on further discussions and agreement with the FDA on the
information needed for an acceptable filing. Sarepta also intends to
have an End-of-Phase II meeting with the agency later this quarter to
discuss the requirements for the Chemistry, Manufacturing, and
Controls (CMC) section of the NDA.
Conference Call Information
Sarepta will hold a conference call to discuss this update today at
8:00 a.m. EDT (5:00 a.m. PDT). 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 35357086. Please
specify to the operator that you would like to join the "Sarepta
Therapeutics Regulatory Update Call." The conference call will be
webcast live under the events section of Sarepta's website at
www.sarepta.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 August 14, 2013 by calling 888.843.7419 or 630.652.3042 and
entering access code 35357086.
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 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
patients usually succumb to the disease in their twenties.
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
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 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 stabilize or
significantly slow the disease process and prolong and improve the
quality of life for patients with DMD.
Sarepta is also developing other PMO-based exon-skipping drug
candidates intended to treat additional 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.sarepta.com.
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 timing of an NDA submission for eteplirsen in
the treatment of DMD; the potential filing and acceptance of an NDA
for eteplirsen; the information necessary for the Agency to make
regulatory determinations and our ability to provide such
information; the potential regulatory approval of eteplirsen
(including under Subpart H Accelerated Approval); and the potential
and timing for manufacturing scale up.
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: the FDA may
determine that our NDA submission for eteplirsen does not qualify for
filing or that substantial additional data is required for
accelerated or other approvals; other decisions by regulatory
authorities; and those identified under the heading "Risk Factors" in
Sarepta's Quarterly Report on Form 10-Q for the quarter ended March
31, 2012, and filed with the Securities and Exchange Commission, and
Sarepta's other filings with the SEC.
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
Sarepta Investor Contact:
Sarepta Media Contact:
Press spacebar to pause and continue. Press esc to stop.