FDA approves Daybue Stix, drug’s powdered formulation, for treating Rett
Mixing with varying liquids before use allows patients to modify strawberry taste
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The U.S. Food and Drug Administration (FDA) has approved Daybue Stix (trofinetide), a dye- and preservative-free powder formulation of Daybue (trofinetide), as a treatment for adults and children, ages 2 and older, with Rett syndrome.
The new formulation will provide Rett patients greater flexibility and choice in dose volume and taste — while maintaining the same efficacy and safety profile as Daybue oral solution — according to a press release from therapy developer Acadia Pharmaceuticals.
Daybue, which won FDA approval in March 2023 as the first U.S. treatment for Rett syndrome, is a sweetened, strawberry-flavored liquid medication. The new formulation, while still strawberry-tasting, has no dyes or preservatives, and can be mixed with varying liquids, such as juice, tea, lemonade, limeade, or liquid hydration, before administration. The volume of Daybue Stix can also be adjusted to a patient’s preference.
“The new formulation gives us an additional option for treatment with Daybue, allowing us to better customize care for our patients,” said Jennifer Martelle Tu, MD, PhD, director of Katie’s Clinic for Rett Syndrome and associate professor of neurology at the University of California, San Francisco Benioff Children’s Hospitals.
The recommended dosage is twice daily, in the morning and evening, based on the patient’s body weight. The therapy, administered orally with or without food or via a feeding tube, will be available on a limited basis starting early next year, and more broadly by June.
Daybue Stix expected to have same safety, efficacy as original med
Before treatment, Daybue Stix powder, available in packets of 5,000 mg, 6,000 mg, or 8,000 mg, must be dissolved in cold to room-temperature water or in beverages.
“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,” Tu said. “We know that this kind of adaptability is something many Rett families have been seeking.”
Among people with Rett syndrome, inflammation and the dysfunction of synapses, the connections between nerve cells, are characteristic abnormalities found in the nervous system.
Trofinetide, the active ingredient in Daybue and Daybue Stix, is a modified form of the naturally occurring protein fragment glypromate, which is thought to reduce inflammation and support synapse health.
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.
Daybue Stix’s efficacy and safety are based on data from a Phase 3 clinical trial, called LAVENDER (NCT04181723), which evaluated the oral solution of Daybue in 187 girls and young women with Rett syndrome. Participants in the trial ranged in age from 5 to 20. Data showed that Daybue significantly eased neurobehavioral symptoms such as fear/anxiety and body rocking or having an expressionless face.
The approval of the new formulation was based on results from a bioequivalence study that showed that the original Daybue oral solution and the Daybue Stix powder for oral solution deliver comparable drug exposure in the body. This demonstrated bioequivalence supports the expectation that Daybue Stix will provide the same efficacy and safety as the oral solution.
Catherine Owen Adams, Acadia’s CEO, noted that Daybue, in both formulations, remains the only approved treatment for Rett in the U.S.
“Daybue … provided thousands of patients an important therapeutic option,” Adams said. “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.”
