trofinetide

Trofinetide is a synthetic tripeptide analogue of the naturally occurring N-terminal tripeptide of insulin-like growth factor 1 (IGF-1), known as glycine-proline-glutamate (GPE). Developed by Neuren Pharmaceuticals and licensed to Acadia Pharmaceuticals, it is the first FDA-approved treatment for Rett syndrome, a rare neurodevelopmental disorder. Trofinetide is thought to exert its therapeutic effects by reducing neuroinflammation and supporting synaptic function, potentially by modulating microglia and astrocyte activity. It is administered as an oral solution and was granted a Rare Pediatric Disease Priority Review Voucher upon its approval in March 2023.

Brand names

Daybue

Other names

trofinetideglycyl-L-2-methylprolyl-L-glutamic acidglycyl-L2-methylprolyl-L-glutamic acidglycyl-L 2-methylprolyl-L-glutamic acid