Sarepta's eteplirsen gets rare pediatric disease designation

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said FDA granted rare pediatric disease designation to eteplirsen (AVI-4658) to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

In June, the company completed the submission of a rolling NDA to FDA for eteplirsen; Sarepta spokesperson Ian Estepan told BioCentury Sarepta expects a filing decision next week. Estepan said Sarepta requested a Priority Review voucher as part of the submission under FDA's rare pediatric disease Priority Review voucher program. The phosphorodiamidate morpholino oligomer (PMO) targeting exon 51 has Orphan Drug designation in the U.S. and EU and Fast Track designation in the U.S. to treat DMD. ...