Acadia Pharmaceuticals Inc., has announced that the U.S. Food and Drug Administration has approved DAYBUE STIX, a powder formulation of trofinetide for oral solution, for treating Rett syndrome in children aged two years and older as well as adults. This new dye- and preservative-free formulation offers the same proven safety and effectiveness as the existing DAYBUE oral solution while providing families and caregivers more flexibility in adjusting dose volume and taste to better suit patient needs.
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“DAYBUE has provided thousands of Rett syndrome patients with a critical therapeutic option. The approval of DAYBUE STIX reflects our commitment to translating scientific advances into meaningful improvements for patients and caregivers,” said Catherine Owen Adams, Chief Executive Officer of Acadia Pharmaceuticals. She added that the company designed this new formulation based on feedback from patients, families, and healthcare providers.
Jennifer Martelle Tu, M.D., Ph.D., Director of Katie’s Clinic for Rett Syndrome and Associate Professor of Neurology at UCSF Benioff Children’s Hospitals in Oakland, highlighted the practical benefits, saying that caregivers can now mix the powder with various water-based liquids to customize taste and dose volume. She emphasized that this kind of adaptability meets a long-standing need for Rett families.
The approval of DAYBUE STIX is supported by results from the LAVENDER study using DAYBUE oral solution in Rett patients. A bioequivalence study confirmed that the powder formulation delivers the same drug exposure as the original oral solution, ensuring comparable safety and effectiveness. DAYBUE STIX will be available in limited quantities in the first quarter of 2026 and more broadly in the second quarter. The original oral solution will continue to remain available.
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Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, occurring in roughly one in 10,000 to 15,000 live births. Patients typically experience normal development for six to 18 months, followed by regression of communication skills and purposeful hand use. Motor abilities may continue to decline throughout life, and most patients require round-the-clock care. The disorder is usually caused by mutations in the MECP2 gene, which may disrupt synaptic communication and contribute to the syndrome’s manifestations. Symptoms often include hand stereotypies, abnormal gait, and cognitive difficulties.
Trofinetide, the active ingredient in both DAYBUE and DAYBUE STIX, is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. While its exact mechanism in Rett syndrome is not fully understood, preclinical studies show it can enhance dendritic branching and support synaptic plasticity, offering a scientific foundation for its therapeutic effects.
This approval represents a significant advancement in care options for individuals with Rett syndrome, providing families with a more flexible treatment while maintaining the clinical benefits of the original formulation.
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Source- businesswire
