Sarepta Therapeutics Announces Intent to Submit an Accelerated Approval Biologics License Application for its Gene Therapy SRP-9001 to Treat Duchenne Muscular Dystrophy
July 29 2022 - 7:00AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced its intent to
submit a Biologics License Application (BLA) seeking accelerated
approval for SRP-9001 (delandistrogene moxeparvovec) to treat
ambulant individuals with Duchenne muscular dystrophy. SRP-9001 is
an investigational gene therapy for Duchenne being developed in
partnership with Roche.
“We are delighted to
confirm that based on the feedback we received following a thorough
and in-depth review, we intend to submit a BLA for our SRP-9001
gene therapy to treat Duchenne muscular dystrophy this fall. We
look forward to a collaborative review commencing this year and
running through the first half of 2023,” said Doug Ingram,
president and chief executive officer, Sarepta Therapeutics.
“Duchenne robs children daily and hourly of their muscle, stealing
them bit by bit from their families and loved ones. Guided by
rigorous science and productive regulatory discussions, our goal is
to move with the urgency desperately needed by the patient
community, and our upcoming BLA filing for SRP-9001 serves that
goal.”
SRP-9001 was granted
Fast Track designation in July 2020, an FDA process designed to
facilitate the development and expedited review of drugs that treat
serious conditions and fill unmet medical needs. In addition
to Fast Track, SRP-9001 has also been granted Rare Pediatric
Disease (RPD) designation in the United States, and Orphan Drug
status in the United States, the European Union, Switzerland and
Japan.
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 benefits of SRP-9001, the
Company’s intent to submit a BLA this fall and the Company
anticipating a collaborative review commencing this year and
running through the first half 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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