Posted by Michael Wonder on 08 Mar 2020
Acacia Pharmaceuticals and Neuren Pharmaceuticals announce rare paediatric disease designation for trofinetide for the treatment of Rett syndrome
3 March 2020 - With no approved treatments in Rett syndrome, FDA decision highlights significant unmet need.
Acacia Pharmaceuticals and Neuren Pharmaceuticals announced today that the U.S. FDA granted Rare Paediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder.
Upon FDA approval of a product with RPD designation, the sponsor may be eligible to receive a priority review voucher, which can be used to obtain FDA review of a new drug application for another product in an expedited period of six months.
