Not content with a stunning launch in the US, Acadia has splashed out $100m to bag the worldwide rights from partner Neuren for Rett syndrome therapy Daybue.

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.

Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.

Axsome’s CRL was not the first from the US FDA for the nearly insoluble NSAID as sponsors try different technologies for delivering its pain relief quickly.