Sarepta Therapeutics has announced the FDA 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.
"We are pleased that the FDA's Office of Orphan Products Development has granted eteplirsen with a Rare Pediatric Disease Designation," said Edward M. Kaye, Sarepta's interim chief executive officer and chief medical officer. 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.”