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Acadia’s Trofinetide Shows Efficacy In Rett Syndrome, But Safety Looks Shaky

 
• By Joseph Haas

Trofinetide could be the first approved drug for Rett patients after hitting both co-primary endpoints in a Phase III trial, but high rates of diarrhea and vomiting raise questions.

Diagnosis Rett syndrome, pills and stethoscope.
Acadia's Phase III data could position trofinetide for first drug approval in Rett syndrome

ACADIA Pharmaceuticals Inc.reported positive Phase III data on 6 December for trofinetide in the rare neurodevelopmental disorder Rett syndrome, which has no approved drug therapy, but analysts are split on whether the candidate’s safety and tolerability profile will hamper its potential approval and uptake.

The San Diego firm said trofinetide, a synthetic analog of the amino-terminal tripeptide of the insulin-like growth factor 1 (IGF-1) hormone that it licensed from Neuren Pharmaceuticals Ltd. in 2018, met its co-primary endpoints in a 187-patient study of girls between the ages of 5 and 20

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