공시 • Apr 08
Acadia Pharmaceuticals Inc Announces DAYBUE STIX Is Now Broadly Available in the United States for the Treatment of Rett Syndrome
Acadia Pharmaceuticals Inc. announced DAYBUE STIX (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide, is now broadly available in the United States for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. The new formulation, approved by the U.S. Food and Drug Administration (FDA) in December 2025, is bioequivalent to the original DAYBUE oral solution, delivering the same efficacy and safety profile, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their DAYBUE treatment. The importance of flexible, patient-centered approaches was reinforced in a recent publication of expert recommendations for real-world use of trofinetide in Rett syndrome. A steering group comprised of experts based at International Rett Syndrome Foundation (IRSF)-designated centers of excellence (COEs) reached consensus recognizing trofinetide oral solution as part of the standard of care for individuals with Rett syndrome. They also aligned on key real-world considerations such as early initiation and sustained use over time. The recommendations also reflect shared perspectives on the need for individualized decision making in clinical practice to help optimize outcomes for patients, families, and caregivers. DAYBUE STIX is a for oral solution powder that caregivers can mix with a variety of water-based liquids such as juice, tea, lemonade, limeade, or liquid hydration so that caregivers have the ability to customize to their loved ones' taste. The product comes in individual packets that are easily portable. The efficacy and safety of DAYBUE STIX is based on the results of the pivotal Phase 3 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. Families interested in exploring this new option should speak with their healthcare provider. Acadia also offers families access to Acadia Connect, a multi-faceted support program that offers a dedicated, experienced support team assisting with financial resources and prescription support to patients and caregivers throughout the DAYBUE treatment journey. The original oral solution formulation approved by the U.S. Food and Drug Administration in 2023 will remain available. DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. Warnings and Precautions: Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was mild or moderate in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE. Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if severe diarrhea occurs or if dehydration is suspected. Vomiting: In a 12-week study, vomiting occurred in 29% of patients treated with DAYBUE and in 12% of patients who received placebo. Patients with Rett syndrome are at risk for aspiration and aspiration pneumonia. Aspiration and aspiration pneumonia have been reported following vomiting in patients being treated with DAYBUE. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if vomiting is severe or occurs despite medical management. Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if significant weight loss occurs. Adverse Reactions: The common adverse reactions (=5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%). Drug Interactions: Effect of DAYBUE and DAYBUE STIX on other Drugs: Trofinetide, a weak inhibitor of CYP3A and an inhibitor of P-gp, can increase the plasma concentrations of CYP3A and/or P-gp substrates (e.g., loperamide), which may increase the risk of adverse reactions associated with these substrates. Closely monitor patients when DAYBUE or DAYBUE STIX is administered concomitantly with sensitive CYP3A and/or P-gp substrates for which a minimal increase in substrate plasma concentration (i.e., drugs with a narrow therapeutic index) may lead to serious adverse reactions. Use in Specific Population: Renal Impairment: DAYBUE and DAYBUE STIX are not recommended for patients with severe renal impairment. DAYBUE is available as an oral solution (200 mg/mL). DAYBUE STIX for oral solution powder is available in 5,000 mg, 6,000 mg, and 8,000 mg packets.