Santhera Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for Santhera's lead orphan drug candidate, idebenone, for the treatment of Duchenne Muscular Dystrophy (DMD). The rare pediatric disease designation supplements the orphan drug designation granted by the FDA for idebenone to treat DMD in February 2007.
"We are pleased that the FDA has granted our request to designate idebenone for the treatment of DMD as a drug for a rare pediatric disease," said Thomas Meier, PhD, Chief Executive Officer of Santhera. "We already have Fast Track designation for idebenone in DMD and, on this basis, will be requesting priority review when we file the planned NDA. The potential to obtain a Rare Pediatric Disease Priority Review Voucher from the FDA, which we could retain for a future clinical development program of our own or sell, could provide additional value for the company in the future."