FDA Approves First Treatment for Rett Syndrome

Posted: 14 March 2023

Neuren Pharmaceuticals announced that its North America partner Acadia Pharmaceuticals received US Food and Drug Administration (FDA) approval of DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Acadia expects DAYBUE to be available by the end of April 2023. DAYBUE is the first and only approved treatment for Rett syndrome.

The announcement by Acadia is attached, which includes details for attending a conference call and webcast by Acadia management on Monday 13 March 2023 at 8:30 am US EDT (11.30 pm AEDT). Neuren will host an investor webinar on Tuesday 14 March 2023, details to be announced separately.

Neuren CEO Jon Pilcher commented: “Many people have shown great determination over the long journey to reach this historic outcome. The greatest has been shown by the Rett syndrome community and I am delighted for them. For Neuren, this is a transforming milestone that places us in a position to make the most of the opportunities ahead of us, as we work with the communities to make a difference in four other neurodevelopmental disorders.”

Find out more.


News & opinion

Member Directory