Neuren Pharmaceuticals Ltd (ASX:NEU) and ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) have been granted a Rare Pediatric Disease (RPD) designation for trofinetide for the treatment of Rett syndrome by the US FDA.
RPD designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the U.S. and primarily in individuals 18 years of age and younger.
Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the US and Orphan Drug Designation for Rett syndrome in Europe.
Debilitating neurological disorder
Rett syndrome is a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life.
The disorder causes problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function.
Rett syndrome occurs worldwide in about one of every 10,000 to 15,000 female births and in the US impacts 6,000 to 9,000 patients.
Drug designed to treat core symptoms of Rett syndrome
Trofinetide is an investigational drug developed by Neuren designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.
In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialisation of trofinetide for Rett syndrome and other indications.
Significance of RPD designation
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.
Under the terms of the license agreement between ACADIA and Neuren, Neuren is eligible to receive one third of the market value of any Rare Pediatric Disease Priority Review Voucher, if awarded by the US FDA upon approval of a New Drug Application for trofinetide.