- Application includes request for Priority Review from the US
FDA
- Sarepta has also submitted the EMBARK postmarketing
requirement to the FDA seeking conversion of the ELEVIDYS
accelerated approval to traditional approval
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today announced
submission of an efficacy supplement to the Biologics License
Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl)
to expand its labeled indication as follows “[ELEVIDYS is indicated
for] the treatment of Duchenne muscular dystrophy (DMD) patients
with a confirmed mutation in the DMD gene.”
The efficacy supplement is supported by results from EMBARK
(Study SRP-9001-301), a global, randomized, double-blind,
placebo-controlled, Phase 3 clinical study in patients with
Duchenne between the ages of 4 through 7 years and data from
ENDEAVOR (Study SRP-9001-103), an open label clinical study in
patients with Duchenne, that is enrolling patients ages 2 years and
older. The supplement was submitted to the U.S. Food and Drug
Administration (FDA) with a request for Priority Review. An
efficacy supplement is a submitted request for a proposed change to
an approved product’s labeling, including adding or modifying an
indication previously filed with the FDA.
Sarepta has also completed the EMBARK postmarketing requirement
(PMR) and submitted the PMR to FDA requesting conversion from
accelerated approval to traditional approval.
“Consistent with our patient commitment, Sarepta moved rapidly
to submit an efficacy supplement to the biologics license
application for ELEVIDYS seeking label expansion to remove age and
ambulation restrictions from the approved indication,” said Doug
Ingram, president and chief executive officer, Sarepta
Therapeutics. “Understanding that Duchenne patients are in a race
against time, we appreciate that the Agency also moved quickly to
engage and provide us with guidance on the process for reviewing
our BLA supplement.”
As part of a collaboration agreement signed in 2019, Sarepta
Therapeutics is working with Roche to transform the future for the
Duchenne community, enabling those living with the disease to
maintain and protect their muscle function. Sarepta is responsible
for regulatory approval and commercialization of ELEVIDYS in the
U.S., as well as manufacturing. Roche is responsible for regulatory
approvals and bringing ELEVIDYS to patients across the rest of the
world. Together, the companies are implementing a comprehensive
joint clinical development plan to maximize the chances of broad
approval and access so that ELEVIDYS can reach as many individuals
with Duchenne as rapidly as possible.
Patients and physicians can access more information about
ELEVIDYS at www.SareptAssist.com or by calling 1-888-727-3782.
About ELEVIDYS (delandistrogene moxeparvovec-rokl)
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose,
adeno-associated virus (AAV) based gene transfer therapy for
intravenous infusion designed to address the underlying genetic
cause of Duchenne muscular dystrophy – mutations or changes in the
dystrophin gene that result in the lack of dystrophin protein –
through the delivery of a shortened transgene that codes for the
targeted production of ELEVIDYS micro-dystrophin in skeletal
muscle. ELEVIDYS is a one-time infusion indicated for the treatment
of ambulatory pediatric patients aged 4 through 5 years with
Duchenne muscular dystrophy (DMD) with a confirmed mutation in the
DMD gene and is approved under accelerated approval based on
expression of ELEVIDYS micro-dystrophin in skeletal muscle observed
in patients treated with ELEVIDYS. Continued approval for this
indication may be contingent upon verification of a clinical
benefit in confirmatory trials. ELEVIDYS has met the full statutory
standards for safety and effectiveness and as such is not
considered investigational or experimental.
In addition to EMBARK, which serves as the postmarketing
confirmatory study, ELEVIDYS has been evaluated in three clinical
studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Accelerated
approval was primarily based on data from SRP-9001-102 and
SRP-9001-103.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATION:
ELEVIDYS is contraindicated in patients with any deletion in
exon 8 and/or exon 9 in the DMD gene.
WARNINGS AND PRECAUTIONS:
Acute Serious Liver Injury:
- Acute serious liver injury has been observed with ELEVIDYS.
Administration of ELEVIDYS may result in elevations of liver
enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically
seen within 8 weeks.
- Patients with preexisting liver impairment, chronic hepatic
condition, or acute liver disease (e.g., acute hepatic viral
infection) may be at higher risk of acute serious liver injury.
Postpone ELEVIDYS administration in patients with acute liver
disease until resolved or controlled.
- Prior to ELEVIDYS administration, perform liver enzyme test and
monitor liver function (clinical exam, GGT, and total bilirubin)
weekly for the first 3 months following ELEVIDYS infusion. Continue
monitoring if clinically indicated, until results are unremarkable
(normal clinical exam, GGT and total bilirubin levels return to
near baseline levels).
- Systemic corticosteroid treatment is recommended for patients
before and after ELEVIDYS infusion. Adjust corticosteroid regimen
when indicated. If acute serious liver injury is suspected, a
consultation with a specialist is recommended.
Immune-mediated Myositis:
- In clinical trials, immune-mediated myositis has been observed
approximately 1 month following ELEVIDYS infusion in patients with
deletion mutations involving exon 8 and/or exon 9 in the DMD gene.
Symptoms of severe muscle weakness including dysphagia, dyspnea and
hypophonia were observed.
- Limited data are available for ELEVIDYS treatment in patients
with mutations in the DMD gene between exons 1 to 17 and exons 59
to 71. Patients with deletions in these regions may be at risk for
a severe immune-mediated myositis reaction.
- Advise patients to contact a physician immediately if they
experience any unexplained increased muscle pain, tenderness, or
weakness, including dysphagia, dyspnea or hypophonia as these may
be symptoms of myositis. Consider additional immunomodulatory
treatment (immunosuppressants [e.g., calcineurin-inhibitor] in
addition to corticosteroids) based on patient’s clinical
presentation and medical history if these symptoms occur.
Myocarditis:
- Acute serious myocarditis and troponin-I elevations have been
observed following ELEVIDYS infusion in clinical trials.
- Monitor troponin-I before ELEVIDYS infusion and weekly for the
first month following infusion and continue monitoring if
clinically indicated. More frequent monitoring may be warranted in
the presence of cardiac symptoms, such as chest pain or shortness
of breath.
- Advise patients to contact a physician immediately if they
experience cardiac symptoms.
Pre-existing Immunity against AAVrh74:
- In AAV-vector based gene therapies, preexisting anti-AAV
antibodies may impede transgene expression at desired therapeutic
levels. Following treatment with ELEVIDYS, all subjects developed
anti-AAVrh74 antibodies.
- Perform baseline testing for the presence of anti-AAVrh74 total
binding antibodies prior to ELEVIDYS administration.
- ELEVIDYS administration is not recommended in patients with
elevated anti-AAVrh74 total binding antibody titers greater than or
equal to 1:400.
Adverse Reactions:
- The most common adverse reactions (incidence ≥ 5%) reported in
clinical studies were vomiting, nausea, liver function test
increased, pyrexia, and thrombocytopenia.
For further information, please see the full Prescribing
Information.
About Sarepta Therapeutics
Sarepta 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 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.
Forward-Looking Statements
This 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, without limitation, statements
relating to our future operations, business plans, priorities,
research and development programs; the potential for our efficacy
supplement to expand the ELEVIDYS label to include Duchenne
patients without restriction to age or ambulatory status; the
potential conversion from accelerated approval to traditional
approval for ELEVIDYS; and our collaboration with Roche.
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 FDA may not approve a supplement to expand the approved label
for ELEVIDYS; we may not be able to comply with all FDA requests in
a timely manner or at all; the possible impact of regulations and
regulatory decisions by the FDA and other regulatory agencies on
our business; our data may not be sufficient for obtaining
regulatory approval; we are subject to uncertainty related to
reimbursement policies; success in preclinical and clinical trials,
especially if based on a small patient sample, does not ensure that
later clinical trials will be successful, and the results of future
research may not be consistent with past positive results or with
advisory committee recommendations, or may fail to meet regulatory
approval requirements for the safety and efficacy of product
candidates; continued approval may be contingent upon verification
of a clinical benefit in confirmatory trials; the commencement and
completion of our clinical trials and announcement of results may
be delayed or prevented for a number of reasons, including, among
others, denial by the regulatory agencies of permission to proceed
with our clinical trials, or placement of a clinical trial on hold,
challenges in identifying, recruiting, enrolling and retaining
patients to participate in clinical trials and inadequate quantity
or quality of supplies of a product candidate or other materials
necessary to conduct clinical trials; 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; we may not be able to execute on our business plans,
including meeting our expected or planned regulatory milestones and
timelines, research and clinical development plans, and bringing
our product candidates to market, for various reasons, many 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 those risks identified under
the heading “Risk Factors” in our most recent Annual Report on Form
10-K for the year ended December 31, 2022, and 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20231222229596/en/
Investor Contact: Ian Estepan, 617-274-4052
iestepan@sarepta.com Media Contact: Tracy Sorrentino,
617-301-8566 tsorrentino@sarepta.com
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