Sarepta Therapeutics Receives Rare Pediatric Disease Designation From FDA

Sarepta Therapeutics (SRPT) announced today that it has received a rare pediatric disease designation from the FDA for its drug Eteplirsen to treat kids with Duchenne Muscular Dystrophy. Eteplirsen has also already received both Orphan drug designation and Fast Track Status by the FDA. This new indication is a voucher that allows the company to gain priority review on a future marketing application to the FDA. 

In that instance FDA could enact it whenever it wants as long as it gives notice to the company that intends to use it. The notice to use such a voucher must be at least 90 days. In addition this voucher can also be sold to another company to use as well. 

Duchenne Muscular Dystrophy is a devastating disease where muscles degenerate at a rapid pace. These patients body's aren't able to produce the proper amount of dystrophin levels to keep muscles intact. Eteplirsen works by restoring dystrophin levels back to normal in these patients. This provides greater quality of life and longer life span for these patients.