FDA rare pediatric disease status for idebenone in Duchenne MD

Swiss specialty pharma company Santhera Pharmaceuticals (SIX: SANN) has received a rare pediatric disease designation from the US Food and Drug Administration for it drug idebenone for the treatment of Duchenne muscular dystrophy.

The rare pediatric disease designation supplements the orphan drug designation granted by the FDA for idebenone to treat DMD in February 2007. The drug is marketed by Santhera under the Raxone and Catena trade names.

"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, chief executive 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," he added.