Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy
November 28 2022 - 7:00AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that the U.S.
Food and Drug Administration (FDA) has accepted the Company's
Biologics License Application (BLA) seeking accelerated approval of
SRP-9001 (delandistrogene moxeparvovec) for the treatment of
ambulant individuals with Duchenne muscular dystrophy. SRP-9001 has
been granted Priority Review by the FDA, with a regulatory action
date of May 29, 2023.
“We are delighted to announce that the FDA has
accepted Sarepta’s BLA for SRP-9001 for filing and priority
review,” said Doug Ingram, president and chief executive officer,
Sarepta Therapeutics. “Duchenne is a relentlessly degenerative and
invariably fatal disease, robbing children of muscle and function
hourly and daily. Our BLA submission for an accelerated approval,
along with the FDA’s acceptance of that BLA for filing and review,
is a tremendously important milestone in our effort to bring a
potentially life-changing gene therapy to Duchenne patients as
rapidly as possible and we look forward to working with the FDA
through the review process.”
SRP-9001 is an
investigational gene therapy for Duchenne being developed in
partnership with Roche. Duchenne is characterized by a mutation in
the dystrophin gene that results in the lack of dystrophin, which
acts as a shock absorber for muscle at the membrane. SRP-9001 is
designed to treat the proximate cause of Duchenne by delivering to
muscle a gene that codes for a shortened, functional form of
dystrophin. In addition to a wealth of pre-clinical evidence, the
BLA for SRP-9001 included efficacy and safety data from Study
SRP-9001-103 (also known as ENDEAVOR), as well as from Studies
SRP-9001-101 and SRP-9001-102, and an integrated analysis across
these three clinical studies comparing functional results to a
propensity-score-weighted external control (EC). In clinical
results from more than 80 treated patients, SRP-9001 has
demonstrated positive results at multiple time points, including
one-, two- and up to four-years after treatment, in addition to
demonstrating a consistent safety profile.
In addition to Studies
SRP-9001-101, SRP-9001-102 and SRP-9001-103, SRP-9001 is also being
studied in EMBARK (Study SRP-9001-301), a global, randomized,
double-blind, placebo-controlled clinical trial of SRP-9001 which
has recruited 125 participants with Duchenne between the ages of 4
to 7. EMBARK is fully enrolled with results expected by the end of
2023. Sarepta has proposed EMBARK as the post-marketing
confirmatory trial for SRP-9001.
About SRP-9001 (delandistrogene
moxeparvovec) SRP-9001 (delandistrogene moxeparvovec) is
an investigational gene transfer therapy intended to deliver
SRP-9001 to muscle tissue for the targeted production of functional
components of dystrophin. Sarepta is responsible for global
development and manufacturing for SRP-9001 and plans to
commercialize SRP-9001 in the United States upon receiving FDA
approval. In December 2019, Roche partnered with Sarepta to combine
Roche’s global reach, commercial presence and regulatory expertise
with Sarepta’s gene therapy candidate for Duchenne to accelerate
access to SRP-9001 for patients outside the United States.
About Duchenne Muscular
Dystrophy Duchenne muscular dystrophy (DMD) is a rare,
fatal neuromuscular genetic disease that occurs in approximately
one in every 3,500-5,000 newborn males worldwide. DMD is caused by
a change or mutation in the gene that encodes instructions for
dystrophin. Symptoms of DMD usually appear in infants and toddlers.
Affected children may experience developmental delays such as
difficulty in walking, climbing stairs or standing from a sitting
position. As the disease progresses, muscle weakness in the lower
limbs spreads to the arms and other areas. Most patients require
full-time use of a wheelchair in their early teens, and then
progressively lose the ability to independently perform activities
of daily living such as using the restroom, bathing and feeding.
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.
About Sarepta
TherapeuticsSarepta is on an urgent mission: engineer
precision genetic medicine for rare diseases that devastate lives
and cut futures short. We hold leadership positions in Duchenne
muscular dystrophy (DMD) and limb-girdle muscular dystrophies
(LGMDs), and we currently have more than 40 programs in various
stages of development. Our vast pipeline is driven by our
multi-platform Precision Genetic Medicine Engine in gene therapy,
RNA and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet
Posting of InformationWe 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.
Forward-Looking StatementsThis press release
contains “forward-looking statements.” Any statements that are not
statements of historical fact may be deemed to be forward-looking
statements. Words such as “believe,” “anticipate,” “plan,”
“expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,”
“possible” and similar expressions are intended to identify
forward-looking statements. These forward-looking statements
include statements relating to the potential benefits of SRP-9001;
our effort to bring a potentially life-changing gene therapy to
Duchenne patients as rapidly as possible; the regulatory action
date of May 29, 2023; our plan to work with the FDA through the
review process; and our expectation that study EMBARK (Study
SRP-9001-301) will serve as the post-marketing confirmatory trial
for SRP-9001 and that we will have results by the end of 2023.
These forward-looking
statements involve risks and uncertainties, many of which are
beyond our control. Known risk factors include, among others: the
possible impact of regulations and regulatory decisions by the FDA
and other regulatory agencies on our business, as well as the
development of our product candidates and our financial and
contractual obligations; that we may not be able to execute on our
business plans and goals, including meeting our expected or planned
regulatory milestones and timelines, clinical development plans,
and bringing our product candidates to market, due to a variety of
reasons, some of which may be outside of our control, including
possible limitations of company financial and other resources,
manufacturing limitations that may not be anticipated or resolved
for in a timely manner, 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 the
COVID-19 pandemic; success in pre-clinical trials and early
clinical trials, especially if based on a small patient sample,
does not ensure that later clinical trials will be successful;
different methodologies, assumptions and applications we use to
assess particular safety or efficacy parameters may yield different
statistical results, and even if we believe the data collected from
clinical trials of our product candidates are positive, these data
may not be sufficient to support approval by the FDA or other
global regulatory authorities; 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, 2021, and 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. For a detailed description of risks and uncertainties
Sarepta faces, you are encouraged to review the SEC filings made by
Sarepta. 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, except as required by law.
Source: Sarepta Therapeutics, Inc.
Investor Contact: Ian Estepan,
617-274-4052iestepan@sarepta.com
Media Contact: Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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