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 approved AMONDYS 45
(casimersen). AMONDYS 45 is an antisense oligonucleotide from
Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform,
indicated for the treatment of Duchenne muscular dystrophy (DMD) in
patients with a confirmed mutation amenable to exon 45 skipping.
This indication is based on a statistically significant increase in
dystrophin production in skeletal muscle observed in patients
treated with AMONDYS 45, which is reasonably likely to predict
clinical benefit for those patients who are exon 45 amenable.
Consistent with the accelerated approval pathway, the continued
approval of AMONDYS 45 may be contingent on confirmation of a
clinical benefit in confirmatory trials.
The ESSENCE trial – a placebo-controlled
confirmatory trial to support the AMONDYS 45 approval – is ongoing
and expected to conclude in 2024.
Although kidney
toxicity was not observed in the clinical studies with AMONDYS 45,
kidney toxicity, including potentially fatal glomerulonephritis,
has been observed after administration of some antisense
oligonucleotides. Kidney function should be monitored in patients
taking AMONDYS 45. In the clinical trial, the most common adverse
reactions observed in at least 20% of patients treated with AMONDYS
45 and at least 5% more frequently than in placebo were (AMONDYS
45, placebo): upper respiratory tract infections (65%, 55%), cough
(33%, 26%), fever (33%, 23%), headache (32%, 19%), joint pain (21%,
10%), and pain in mouth and throat (21%, 7%).
“This is an important
day for Sarepta and, far more importantly, for the patients that we
serve. After years of scientific commitment, investment and
development, the approval of AMONDYS 45, Sarepta’s third approved
RNA therapy, offers treatment to the 8% of the DMD community who
have a confirmed exon 45 amenable mutation,” said Doug Ingram,
president and chief executive officer, Sarepta. “Along with our
other approved RNA therapies, we can now offer treatment options
for nearly 30% of Duchenne patients in the U.S. And our commitment
to bring therapies to the greatest percentage of the DMD community
as soon as possible continues.”
“Decades of research
and commitment have fueled and now accelerate our progress towards
new treatments for Duchenne,” said Marissa Penrod, founder of Team
Joseph and parent of an 18-year old with Duchenne. “The
extraordinary diligence and persistence of the Duchenne community –
patients and families, clinicians and researchers – have led us to
today’s approval, where we now have exon-skipping treatments for
almost a third of those with Duchenne.”
AMONDYS 45 is priced at parity with Sarepta’s
other approved exon-skipping treatments. Patients and physicians
can access more information at www.SareptAssist.com or by calling
1-888-727-3782.
About AMONDYS 45AMONDYS 45
(casimersen) is an antisense oligonucleotide indicated for the
treatment of Duchenne muscular dystrophy in patients who have a
confirmed mutation of the DMD gene that is amenable to exon 45
skipping. AMONDYS 45 uses Sarepta’s proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to
bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or
“skipping,” of this exon during mRNA processing in patients with
genetic mutations that are amenable to exon 45 skipping. Exon
skipping is intended to allow for production of an internally
truncated dystrophin protein.
AMONDYS 45 is approved under accelerated review
based on an increase in dystrophin production in skeletal muscle of
patients amenable to exon 45 skipping. Continued approval may be
contingent upon verification of a clinical benefit in confirmatory
trials.
AMONDYS 45 has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
Important Safety Information for AMONDYS
45Kidney toxicity was observed in animals who received
casimersen. Although kidney toxicity was not observed in the
clinical studies with AMONDYS 45, kidney toxicity, including
potentially fatal glomerulonephritis, has been observed after
administration of some antisense oligonucleotides. Kidney function
should be monitored in patients taking AMONDYS 45. Because of the
effect of reduced skeletal muscle mass on creatinine measurements,
creatinine may not be a reliable measure of kidney function in DMD
patients. Serum cystatin C, urine dipstick, and urine
protein-to-creatinine ratio should be measured before starting
AMONDYS 45. Consider also measuring glomerular filtration rate
using an exogenous filtration marker before starting AMONDYS 45.
During treatment, monitor urine dipstick every month, and serum
cystatin C and urine protein-to-creatinine ratio (UPCR) every three
months. Only urine expected to be free of excreted AMONDYS 45
should be used for monitoring of urine protein. Urine obtained on
the day of AMONDYS 45 infusion prior to the infusion, or urine
obtained at least 48 hours after the most recent infusion, may be
used. Alternatively, use a laboratory test that does not use the
reagent pyrogallol red, as this reagent has the potential to cross
react with any AMONDYS 45 that is excreted in the urine and thus
lead to a false positive result for urine protein.
If a persistent increase in serum cystatin C or
proteinuria is detected, refer to a pediatric nephrologist for
further evaluation.
Adverse reactions observed in at least 20% of
patients treated with AMONDYS 45 and at least 5% more frequently
than in the placebo group were (AMONDYS 45, placebo): upper
respiratory tract infections, including upper respiratory
infection, pharyngitis, nasopharyngitis and rhinitis (65%, 55%),
cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%),
arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%).
Other adverse reactions that occurred in at
least 10% of patients treated with AMONDYS 45, and that were
reported at a rate at least 5% more frequently in the AMONDYS 45
group than in the placebo group, were: ear pain, nausea, ear
infection, post-traumatic pain, and dizziness and
light-headedness.
For further information, please see the full
Prescribing Information.
About Sarepta
TherapeuticsAt Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us
on Twitter, LinkedIn, Instagram and Facebook.
Forward-Looking StatementThis
press release contains "forward-looking statements." 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
immediate commencement of commercial distribution of AMONDYS 45 in
the U.S.; AMONDYS 45’s continued approval potentially being
contingent upon confirmation of a clinical benefit in confirmatory
trials; the expectation to conclude the ESSENCE trial by 2024;
AMONDYS 45’s potential to treat 8% of the DMD community who have a
confirmed exon 45 amenable mutation; Sarepta being able to offer
treatment options for nearly 30% of Duchenne patients in the U.S.;
the potential benefits and risks of AMONDYS 45; our commitment to
bring therapies to the greatest percentage of the DMD community as
soon as possible; and exon skipping’s intention to allow for
production of an internally truncated dystrophin protein.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: the planned commercial
launch in the U.S. for AMONDYS 45 may not be successful for various
reasons including the actual market size and drug supply needed may
not be consistent with the company’s expectations and its executed
commercial readiness plans, the degree to which AMONDYS 45 is
accepted by patients and prescribed by physicians, manufacturing
limitations that may not be anticipated or resolved for in a timely
manner or at all, the efficiency of our manufacturing, sales,
distribution and specialty pharmacy network in getting AMONDYS 45
to the market, the response to COVID-19 and future economic,
competitive, reimbursement and regulatory conditions that could
negatively impact the commercial launch of AMONDYS 45; we may not
be able to comply with all FDA post-approval commitments and
requirements with respect to our products in a timely manner or at
all; the results of our ongoing research and development efforts
and clinical trials for our products and product candidates may not
be positive or consistent with prior results or demonstrate a safe
treatment benefit or support an NDA or a BLA filing, positive
advisory committee recommendation or marketing approval by the FDA
or other regulatory authority; we may not be able to execute on our
business plans including meeting our expected or planned regulatory
milestones and timelines, clinical development plans and bringing
our product candidates to market, including the commercialization
of AMONDYS 45, for various reasons, including factors outside of
our control, such as possible limitations of company financial and
other resources, manufacturing limitations that may not be
anticipated or resolved for in a timely manner or at all, COVID-19
and regulatory, court or agency decisions, such as decisions by the
United States Patent and Trademark Office with respect to patents
that cover our product and product candidates; 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,
2019, 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 Sarepta's 2019 Annual Report on Form 10-K and
most recent Quarterly Report on Form 10-Q filed with the SEC as
well as other 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.
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.
Source: Sarepta Therapeutics, Inc.
Sarepta Contacts:
Investors:Ian Estepan, 617-274-4052,
iestepan@sarepta.comMedia:Tracy
Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
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