ACADIA Pharmaceuticals Inc. obtained US Food and Drug Administration approval for Daybue (trofinetide), its synthetic analog of the amino terminal tripeptide of IGF-1, as the first drug therapy for Rett syndrome on 10 March and expects the product will be available by late April. The company also said it is happy with the indication and safety language in labeling given the drug’s high incidence of diarrhea in clinical studies.
Acadia’s Daybue Gets First Approval In Rett Syndrome
Daybue is the first drug approved to treat the rare neurodevelopmental disorder Rett syndrome, despite high clinical trial incidence of diarrhea. Product labeling includes clinician guidance for the side effect.
