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Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date

The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. PPMD has been in touch with Sarepta and our coalition partners regarding next steps. We will update the community as we learn more.

 

PPMD continues to believe eteplirsen is safe and effective and suitable for accelerated approval, and we hope such a decision from FDA is forthcoming. 

 

The Duchenne community, in benefit-risk studies led by PPMD, has provided quantifiable data regarding our highest priority for emerging treatments -- stopping or slowing the progression of Duchenne. Those with experience on eteplirsen have communicated to the agency about the benefits they see and experience, and the scientific and clinical experts in this field have weighed in with their full support of this therapeutic option.  

 

We remain hopeful that the FDA is using this time to ensure that the collective voice of the community is being taken into account in this decision.

Read the Press Release from Sarepta: 

Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 25, 2016-- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has notified the Company that they are continuing their review and internal discussions related to our pending NDA for eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. The FDA has communicated that they will continue to work past the PDUFA goal date and strive to complete their work in as timely a manner as possible.

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 rare, infectious and other diseases. For more information, please visit us at www.sarepta.com.

About Eteplirsen

Eteplirsen is designed to address the underlying cause of DMD by restoring the dystrophin messenger RNA (mRNA) reading frame, thus enabling the production of a shorter, functional form of the dystrophin protein. Eteplirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Approximately 13 percent of the DMD population is amenable to exon 51 skipping. Data from clinical studies of eteplirsen in DMD patients have demonstrated a consistent safety and tolerability profile and have also shown measurable dystrophin protein expression. Promoting the synthesis of a shorter dystrophin protein is intended to slow the decline of ambulation and mobility seen in DMD patients. There currently is no approved treatment in the United States for DMD and eteplirsen has not been approved by the FDA or any regulatory authority for the treatment of 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 3,500-5,000 males 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 FDAnot completing its review of the eteplirsen NDA by the PDUFA date of May 26, 2016, additional review and internal FDA discussions relating to the NDA, and the timing for the FDA completing its work and reaching a decision with respect to the Company’s eteplirsen NDA. 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 its decision on the eteplirsen NDA or may not provide marketing approval for eteplirsen; we may not be able to comply with all FDA requests, including with respect to our eteplirsen NDA submission and the addendums we have submitted to the FDA or with respect to our ongoing or planned clinical trials, in a timely manner or at all; we may not be able to complete clinical trials required by the FDA for approval of our products or any submissions made in connection with our pipeline of product candidates; the results of our ongoing research and development efforts and clinical trials for our product candidates including eteplirsen and our technologies may not be positive or consistent with prior results or demonstrate a safe treatment benefit or support an NDA 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 planned commercialization of eteplirsen, for various reasons, including factors outside of the Company’s control, including 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, 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 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, 2015 or Quarterly Report on Form 10-Q for the quarter ended March 31, 2016 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta 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 theSEC. 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

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.

Link to Press Release

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