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.

The PureTech Health-founded entity believes it has made a significant step forward in the treatment of schizophrenia with its muscarinic-receptor acting agent KarXT, which could offer a completely new way to address all three symptom domains of the disease.

The endpoint used in the AVATAR trial was different than the one investors had expected; management said the change was made to satisfy the US FDA.

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.