Acadia Pharmaceuticals (NASDAQ:ACAD) announced today that the U.S. Food and Drug Administration (FDA) has approved DAYBUE® STIX (trofinetide) for oral solution, a new dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in both adult and pediatric patients aged two years and older.
The approval provides patients with a new, flexible treatment option, offering both a choice in dosing volume and an improved taste compared to the original oral solution.
The efficacy and safety of DAYBUE STIX are based on the results of the LAVENDER™ study, which evaluated the original DAYBUE oral solution in patients with Rett syndrome.
The approval was supported by data from a bioequivalence study, which demonstrated that DAYBUE STIX provides comparable exposure to the original oral solution, confirming that the new formulation will offer the same established efficacy and safety profile.
DAYBUE STIX is expected to be available on a limited basis starting in the first quarter of 2026, with broader availability anticipated in the second quarter of 2026.
The original oral solution formulation will remain available to patients.
This approval marks a significant step forward in providing greater flexibility for patients and their caregivers in managing Rett syndrome, offering a well-established treatment with the convenience of a new formulation.