Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51
09/19/16 1:40 PM EDT
-- EXONDYS 51™ the first DMD treatment approved in the US, targets dystrophin deficiency, the underlying cause of Duchenne --
--U.S. Commercial Launch planned to commence immediately--
--Conference call Scheduled for
“Today’s accelerated approval of EXONDYS 51 represents a major milestone
in the treatment of Duchenne Muscular Dystrophy for patients amenable to
skipping exon 51 by targeting the underlying genetic cause of the
disease – the lack of the dystrophin protein,” said
The underlying cause of Duchenne muscular dystrophy is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Certain genetic mutations in DMD involve the deletion of exons, which interrupt proper translation of the genetic code into protein.
Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder affecting an estimated one in approximately every 3,500 – 5,000 males born worldwide. It is estimated that up to thirteen percent of people with DMD have mutations addressable by EXONDYS 51.
Patients and physicians can access information by visiting www.SareptAssist.com or calling 1-888-727-3782.
The Company will be hosting a conference call at
About Duchenne Muscular Dystrophy (DMD)
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. DMD is estimated to affect approximately one in every 3,500-5000 males born 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 death usually occurs before the age of 30.
About EXONDYS 51™
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.
Important Safety Information
- Adverse reactions observed in patients (N=8) treated with 30 or 50 mg/kg/wk of EXONDYS 51 with incidence ≥ 25% and higher than in the placebo group (N=4) (Study 1) were: balance disorder (38%), vomiting (38%) and contact dermatitis (25%). The most common adverse reactions were balance disorder and vomiting.
- The following events were reported in ≥ 10% of patients treated with EXONDYS 51 for up to 208 weeks (N=88) and occurred more frequently than placebo in a controlled trial for 24 weeks (Study 1): vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.
- There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including EXONDYS 51, designed to skip exon 51 and approved under the accelerated approval pathway. For more information, please visit us at www.sarepta.com.
Forward Looking Statements
This press release contains "forward-looking statements" within the
meaning of the safe harbor provisions of the
These forward-looking statements involve risks and uncertainties,
many of which are beyond Sarepta's control or unknown. Known risk
factors include, among others: the planned commercial launch in the US
for EXONDYS 51 may not be successful in part or at all 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 EXONDYS 51 is accepted
by patients and prescribed by physicians, the efficiency of our
manufacturing, sales, distribution and specialty pharmacy network in
getting Exondys51 to the market and future economic, competitive,
reimbursement and regulatory conditions that could negatively impact the
commercial launch of EXONDYS 51; the confirmatory and other studies for
Exondys51 may not yield data consistent with prior results or
demonstrate a benefit that supports continued or full regulatory
approval; we may not be able to complete clinical trials required by the
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
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