Sarepta Therapeutics Inc (NASDAQ:SRPT) announced that the FDA has granted accelerated approval for EXONDYS 51™ (eteplirsen) as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. This indication is based on an increase in dystrophin in skeletal muscles observed in some patients treated with EXONDYS 51. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. The most common adverse reactions compared to a placebo group were vomiting (38%) and balance disorder (38%) with contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection also reported more frequently than placebo (≥ 10%).
“Today’s accelerated approval of EXONDYS 51 represents a major milestone in the treatment of Duchenne Muscular Dystrophy for patients amenable to skipping exon 51 by targeting the underlying genetic cause of the disease – the lack of the dystrophin protein,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We are grateful to the many patients and investigators who participated in EXONDYS 51’s clinical studies. EXONDYS 51 represents the culmination of many years of work across our entire organization and the Duchenne community to address a critical unmet need by bringing this novel medicine to patients. We will continue to leverage what we have learned from EXONDYS 51 to facilitate future development of potential new treatments targeting additional exons with the goal of one day treating all DMD patients amenable to exon skipping.” (Original Source)
Shares of Sarepta jumped nearly 90% to $53.41 as of this writing. SRPT has a 1-year high of $56.18 and a 1-year low of $8. The stock’s 50-day moving average is $26.68 and its 200-day moving average is $20.82.
On the ratings front, Sarepta has been the subject of a number of recent research reports. In a report released today, Janney Montgomery Scott analyst Debjit Chattopadhyay upgraded SRPT to Buy. Separately, Oppenheimer’s Michelle Gilson reiterated a Buy rating on the stock and has a price target of $60.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Debjit Chattopadhyay and Michelle Gilson have a total average return of -1.2% and 16.2% respectively. Chattopadhyay has a success rate of 44% and is ranked #3303 out of 4163 analysts, while Gilson has a success rate of 64% and is ranked #741.
Overall, 3 research analysts have rated the stock with a Sell rating, 5 research analysts have assigned a Hold rating and 5 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 $24.50 which is -15.0% under where the stock opened today.
Sarepta Therapeutics, Inc. is a biopharmaceutical company, which is engaged in the discovery and development of unique RNA-based therapeutics for the treatment of rare and infectious diseases. The company is primarily focused on rapidly advancing the development of its potentially disease modifying duchenne muscular dystrophy drug candidates.