CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016. There has been no formal communication to the company or Federal Register notice of a confirmed advisory committee meeting date.
It is estimated that DMD affects approximately one in every 3,500 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
“We have been verbally informed by the FDA that we have a tentative date of January 22, 2016 for our advisory committee review of eteplirsen and our PDUFA date of February 26, 2016, remains unchanged.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We look forward to discussing the efficacy and safety data included in our NDA submission for eteplirsen with the committee, with the ultimate goal of bringing treatment to more patients with Duchenne.”
About Sarepta Therapeutics
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases, such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.
About Eteplirsen
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% 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.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. 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 potential date for a meeting of an FDA advisory committee to review the NDA for eteplirsen and the applicable PDUFA date, the potential market for eteplirsen, Sarepta’s planned discussions on efficacy and safety data included in the NDA submission with the Committee and Sarepta’s ultimate goal of bringing treatment to more patients with Duchenne. Forward-looking statements also include those regarding Sarepta’s future business developments and actions and the timing of the same.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the FDA may further delay or cancel the tentative advisory committee meeting, the results of our ongoing research and development efforts and clinical trials for eteplirsen and our other product candidates may not be positive or consistent with prior results or demonstrate a safe treatment benefit, there may be delays in Sarepta's projected regulatory and development timelines relating to the eteplirsen NDA and plans for commercializing eteplirsen and developing Sarepta's other product candidates for various reasons including possible limitations of Sarepta's financial and other resources; Sarepta may not be able to successfully complete its planned commercialization of eteplirsen or continue developing its product candidates as planned for a variety of reasons including due to regulatory, court or agency decisions, such as decisions by the USPTO with respect to patents that cover Sarepta's product candidates, scale-up of manufacturing may not be successful, and any or all of Sarepta's product candidates may fail in development or may not receive required regulatory approvals for commercialization; and those risks identified under the heading “Risk Factors” in Sarepta's 2014 Annual Report on Form 10-K or and most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2015 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 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 SEC. 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 Information
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