Sarepta Therapeutics Inc (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) date for eteplirsen has been extended to May 26, 2016. The rescheduled date for the Peripheral and Central Nervous System Advisory Committee meeting has not yet been determined.
The FDA notified Sarepta that its January 8, 2016 submission of 4-year clinical effectiveness data, which included additional six minute walk test (6MWT) and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. TheFDA stated that the PDUFA goal date has been extended by three months to allow for a full review of the submission. As described in the Sarepta Advisory Committee Briefing Document Addendum, the principal basis for establishing the effectiveness of eteplirsen is a comparison of patients in Study 201/202 to a historical control group.
“While our primary goal is to bring treatment to patients with Duchenne as quickly as possible, we appreciate the efforts of the FDA to conduct a complete review of all of the data supporting our NDA and we remain committed to working closely with them throughout the remainder of the regulatory process”, said Edward Kaye, Sarepta’s interim chief executive officer and chief medical officer.
The FDA has previously granted eteplirsen Priority Review status, which is designated for drugs that provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to eteplirsen, as well Orphan Drug Designation and Fast Track Status.
It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13 percent of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.(Original Source)
Shares of Sarepta closed last Friday at $12.22. SRPT has a 1-year high of $41.97 and a 1-year low of $11.10. The stock’s 50-day moving average is $25.25 and its 200-day moving average is $31.27.
On the ratings front, Sarepta has been the subject of a number of recent research reports. In a report issued on January 20, Oppenheimer analyst Christopher Marai maintained a Buy rating on SRPT, with a price target of $45, which represents a potential upside of 268.2% from where the stock is currently trading. Separately, on January 19, RBC’s Simos Simeonidis reiterated a Hold rating on the stock and has a price target of $15.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Christopher Marai and Simos Simeonidis have a total average return of 9.2% and -32.0% respectively. Marai has a success rate of 47.2% and is ranked #121 out of 3584 analysts, while Simeonidis has a success rate of 17.4% and is ranked #3550.
Overall, 5 research analysts have assigned a Hold rating and 4 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 $42.50 which is 247.8% 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.