Acadia Pharmaceuticals Inc. and Neuren Pharmaceuticals (ASX:NEU) announced today that the US Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder.
Upon FDA approval of a product, 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.
They are continuing to c enroll patients in their Phase 3 LAVENDER study with results expected in 2021.
Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the U.S. and Orphan Drug Designation for Rett syndrome in Europe.
Shares in Neuren Pharmaceuticals (ASX: NEU) are trading flat at $1.93.