FDA approves Daybue Stix (trofinetide) for oral solution for the treatment of Rett syndrome – Acadia Pharmaceuticals

Acadia Pharmaceuticals announced that the FDA has approved Daybue Stix (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. The new formulation is expected to deliver the same efficacy and safety profile of Daybue oral solution, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their Daybue treatment.

The efficacy and safety of Daybue Stix is based on the results of the LAVENDER study with Daybue oral solution in patients with Rett syndrome. The approval of this new formulation was informed by the results of a bioequivalence study, which demonstrated that both original Daybue oral solution and the new  Daybue Stix for oral solution powder formulation provide comparable exposure. This confirmed bioequivalence means patients can expect the same efficacy and safety established by the oral solution formulation when using  Daybue Stix.

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.

Daybue and Daybue Stix are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

“Daybue , the first and only approved treatment for Rett syndrome, has provided thousands of patients an important therapeutic option. With the approval of  Daybue Stix, we are building on our commitment to turn scientific promise into meaningful innovation by introducing a new formulation informed by invaluable feedback from patients, caregivers and healthcare providers.” – Catherine Owen Adams, Acadia’s Chief Executive Officer.

“The new formulation gives us an additional option for treatment with DAYBUE, allowing us to better customize care for our patients. Daybue Stix is a powder for oral solution that caregivers can mix with a variety of water-based liquids providing flexibility to modify the taste and volume of their loved-one’s dose. We know that this kind of adaptability is something many Rett families have been seeking.” – Jennifer Martelle Tu, M.D., Ph.D., Director of Katie’s Clinic for Rett Syndrome and Associate Professor of Neurology, UCSF Benioff Children’s Hospitals, Oakland.