Sarepta Therapeutics Inc (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015. Eteplirsen, the Company’s lead drug candidate, targets the underlying cause of Duchenne muscular dystrophy and is designed to enable the production of a functional internally truncated dystrophin protein in patients with mutations amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation targeted by Eteplirsen/exon 51 skipping.
“The completion of our NDA submission for eteplirsen represents the culmination of the efforts of our employees, investigators, clinical trial sites, and most importantly the patients and families of the Duchenne community,” said Edward M. Kaye, interim chief executive officer and chief medical officer. “We look forward to working with the FDA during the regulatory process in pursuit of our goal of bringing eteplirsen to patients amenable to exon 51 skipping, while maintaining our organizational focus on advancing our PMO technology to target other DMD subpopulations amenable to exon-skipping as quickly as possible.”
The NDA submission includes a request for Priority Review. Previously, eteplirsen has been granted Orphan and Fast Track status by the FDA.
The rolling submission of the NDA began on May 20, 2015, after the completion of a pre-NDA meeting with the FDA held on May 19, 2015. (Original Source)
Shares of Sarepta Therapeutics Inc. closed last Friday at $31.26 . SRPT has a 1-year high of $33.16 and a 1-year low of $11.33. The stock’s 50-day moving average is $25.27 and its 200-day moving average is $16.44.
On the ratings front, Sarepta Therapeutics Inc. has been the subject of a number of recent research reports. In a report issued on June 9, RBC analyst Simos Simeonidis reiterated a Hold rating on SRPT, with a price target of $20, which represents a potential downside of 36.0% from where the stock is currently trading. Separately, on June 7, Roth Capital’s Debjit Chattopadhyay reiterated a Buy rating on the stock and has a price target of $45.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Simos Simeonidis and Debjit Chattopadhyay have a total average return of -11.0% and 31.9% respectively. Simeonidis has a success rate of 32.3% and is ranked #3570 out of 3681 analysts, while Chattopadhyay has a success rate of 67.4% and is ranked #18.
In total, 4 research analysts have assigned a Hold rating and 7 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $35.00 which is 12.0% above where the stock closed last Friday.
Sarepta Therapeutics Inc is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases.