Sarepta Therapeutics Receives Rare Pediatric Disease Designation From FDA for Eteplirsen for the Potential Treatment of Duchenne Muscular Dystrophy
08/21/15 8:30 AM EDT
"We are pleased that the
About Rare Pediatric Disease Designation
About Sarepta Therapeutics
Eteplirsen is Sarepta's lead drug candidate and is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys 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.
This press release contains statements that are forward looking. 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,” “may,”
“intends,” “prepares,” “looks,” “potential,” “possible” and similar
expressions are intended to identify forward-looking statements. These
forward-looking statements include statements relating to eteplirsen
receiving rare pediatric disease designation from the
These forward-looking statements involve risks and uncertainties,
many of which are beyond Sarepta’s control. 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: we may never receive a priority review
voucher as a result of the rare pediatric disease designation for
eteplirsen or, if we do receive a voucher, we may not be able to use it
to benefit any of our product candidates or transfer it for various
reasons, such as the expiration or termination of the priority review
voucher program or failure to obtain regulatory approval of eteplirsen;
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. You should not place undue reliance on
forward-looking statements. Sarepta does not undertake any obligation to
publicly update its forward-looking statements based on events or
circumstances after the date hereof, except to the extent required by
applicable law or
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