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Neuren and Acadia’s trofinetide wins FDA rare pediatric disease designation for Rett syndrome as phase III testing advances

March 10, 2020
By Tamra Sami
PERTH, Australia – The FDA granted Melbourne-based Neuren Pharmaceuticals Ltd. and San Diego, California-based Acadia Pharmaceuticals Inc. a rare pediatric disease designation for trofinetide for treatment of Rett syndrome, a rare neurodevelopmental congenital central nervous system disorder.
BioWorld Asia Drugs Regulatory Neurology/psychiatric Australia FDA