Sarepta's eteplirsen gains Rare Pediatric Disease Designation from FDA

US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) says that the US Food and Drug Administration has granted Rare Pediatric Disease Designation for eteplirsen, a potential treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to skipping exon 51.

The Rare Pediatric Disease Designation supplements the Orphan Drug Designation and Fast Track Status previously granted by the FDA for eteplirsen. Sarepta’s shares were down 1.34% to close at $33.86 on Friday, but the stock is up 109.27% over the past 200 days, analysts have noted.

"We are pleased that the FDA's Office of Orphan Products Development has granted eteplirsen with a Rare Pediatric Disease Designation," said Edward Kaye, Sarepta's interim chief executive and chief medical officer, adding: “We appreciate that FDA has created the Rare Pediatric Disease Priority Review Voucher program to foster development of treatments for rare pediatric diseases, a core focus for the company, and we hope it leads to expedited treatments for children who desperately need them.”