DAYBUE trofinetide
Drug Class
Synthetic analog of the amino-terminal tripeptide of IGF-1IGF-1 氨基末端三肽的合成类似物
Mechanism
Trofinetide is a synthetic analog of glypromate (GPE) that is thought to reduce neuroinflammation, restore synaptic function, and improve dendritic branching in the brain.曲芬尼肽是甘氨酰-脯氨酰-谷氨酸 (GPE) 的合成类似物，被认为可以减少神经炎症，恢复突触功能，并改善大脑中的树突分叉。
Approved Indication
Treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.用于治疗2岁及以上成人和儿童患者的雷特 (Rett) 综合征。
What This Means For Patients
Daybue is a prescription medication for the treatment of Rett syndrome, a rare neurodevelopmental disorder. It is an oral solution taken twice daily to target core neurobehavioral symptoms. It is accessible to patients in China at the Hainan Boao Lecheng pilot zone under special import regulations.Daybue（曲芬尼肽）是获美国FDA批准用于治疗雷特（Rett）综合征（一种罕见的神经发育障碍）的处方药物。它是一种每日服用两次的口服液，针对核心神经行为症状。该药物已通过海南博鳌乐城的特许进口渠道引入。
Pivotal Clinical Evidence
- LAVENDER — Neul et al., Nat Med 2023 (NCT04181723) — DOI
Sources
- https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217026s000lbl.pdf
- https://www.ema.europa.eu/en/medicines/human/summaries-opinion/daybue
- https://www.acadia.com/media/news-releases/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients/
This page provides regulatory and mechanism-of-action information for reference only. It is not medical advice, a treatment recommendation, or a guarantee of outcome. Availability is subject to individual clinical review.