FDA approves oral solution for Neuren's Rett syndrome treatment

The news: Biopharmaceutical company Neuren Pharmaceuticals has announced that its US distributor, Acadia Pharmaceuticals, has received US Food and Drug Administration (FDA) approval of its 'Daybue Stix' oral solution.

The context: Daybue Stix is a new powder formulation of the prescription medication trofinetide, used to treat Rett syndrome, a rare genetic neurodevelopmental disorder that affects brain development, in patients two years and older. It is the first and only FDA-approved treatment for the condition.

Acadia expects Daybue Stix to be available on a limited basis starting in the first quarter of 2026.

What they said: "The Neuren team is excited about the approval of this new treatment option for Rett syndrome. Caregivers can mix Daybue Stix with a variety of water-based liquids providing the flexibility to modify the taste and volume of their dose. We look forward to seeing the impact as Daybue Stix becomes more broadly available during 2026," Neuren Pharmaceuticals CEO Jon Pilcher said.