Sarepta Therapeutics, Inc. (NASDAQ: SRPT)
SRPT, a relatively young biopharmaceutical company, has been seeking FDA approval for its (DMD) Duchenne Muscular Dystrophy drug candidate and has been nothing less of explosively volatile in recent trading grabbing the attention of traders and investors. The most recent activity was sparked back on May 25 when the FDA chose to “delay” its decision on Sarepta’s DMD drug, sending shares of SRPT lower by over 25%.
Analysts at Piper Jaffray had the following comments on SRPT.
This morning the FDA finalized new procedures for expanded access for unapproved drugs. The agency streamlined the Individual Patient Expanded Access IND to simplify the process. FDA Commissioner Califf mentioned the importance of accessing investigational treatments for serious disease where there are no other options. In our view, this could enable broader access to eteplirsen that the DMD community is desperate for, without forcing the agency to grant an approval at this time. The FDA recently indefinitely delayed regulatory decision on eteplirsen beyond the May 26 PDUFA date. We continue to believe Accelerated Approval is unlikely based on the current data set. Importantly, while sponsors can seek FDA authorization to charge for drugs in expanded access, it may only recoup direct manufacturing costs (not development or regulatory costs), limiting the sales potential. Shares of SRPT are trading lower. We reiterate our Underweight rating and $6 price target.
TheFly.com also had a few notable additions from earlier today on SRPT.
The U.S. Food and Drug Administration announced it has finalized its efforts to streamline the process used by physicians to request expanded access, often called “compassionate use,” to investigational drugs and biologics for their patients. In conjunction, the FDA is releasing the final Individual Patient Expanded Access Investigational New Drug Application – Form FDA 3926. “The new form can be used by physicians to request expanded access to investigational drugs for individual patients who suffer from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative therapy is available. It is much shorter than the form previously used for individual patient expanded access requests and is designed specifically for these requests. It should take about 45 minutes for a physician to complete the new form. Along with the new form we are also releasing step-by-step instructions on how to complete it,” the agency stated. Weighing in on the announcement, TheStreet’s biotech reporter Adam Feuerstein said “this compromise won’t be financially friendly” to Sarepta (SRPT) “or its stock price.” Sarepta shares are down nearly 9% in pre-market trading.
About Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc., incorporated on June 5, 2013, is a biopharmaceutical company. The Company is focused on the discovery and development of ribonucleic acid (RNA)-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is engaged in the development of pharmaceutical products on its own behalf or in collaboration with others. The Company, through its platform technologies, targets a range of diseases and disorders through RNA-targeted mechanisms of action. The Company is primarily focused on advancing the development of its disease-modifying Duchenne Muscular Dystrophy (DMD) drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. The Company is also developing therapeutics using its technology for the treatment of drug-resistant bacteria and infectious, rare and other human diseases.