What You Need to Know About Sarepta Therapeutics Inc (SRPT)’s Q4 Update

The FDA’s stance will hinge largely on how it views the safety profile of eteplirsen and how confident it is that Sarepta can manufacture the drug with high quality levels. Although clinical studies to date have only included a small number of patients, company management emphasized that by later this year several patients will have accumulated two years of taking eteplirsen at high dosages. They clearly hope that the pressing need for a treatment for DMD will help sway the FDA.

Recent concerns have been raised about GlaxoSmithKline plc (ADR) (NYSE:GSK)‘s DMD drug, drisapersen. In particular, there were reports about the drug resulting in kidney toxicity. Garabedian alluded to FDA concerns about kidney toxicity and expressed confidence in the safety profile of eteplirsen in that regard.

Another thing that we still don’t know is if Sarepta Therapeutics Inc (NASDAQ:SRPT) will seek Breakthrough Therapy designation for eteplirsen. This designation allows for a much quicker review and approval process. The company thinks that eteplirsen could be a good candidate and is considering pursuing the designation, but a decision won’t be made until after talks with the FDA.

Looking ahead
While shares were down around 4% in intraday trading, the outlook for Sarepta hasn’t changed. If the discussions with the FDA go well later this month and the company pursues accelerated approval, I expect the stock will enjoy a nice bump. At this point, I think it’s likely that this scenario will play out.

Of course, that doesn’t mean that the FDA will go along with accelerated approval. There could be enough doubts about safety to cause the agency to proceed with caution. If that happens, the stock will undoubtedly take a hit. Regardless of the accelerated approval path, though, I like the prospects for Sarepta over the long run.

The article What You Need to Know About Sarepta’s Q4 Update originally appeared on Fool.com and is written by Keith Speights.

Fool contributor Keith Speights has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned.

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