DAYBUE trofinetide

Drug Class

Synthetic analog of the amino-terminal tripeptide of IGF-1

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.

Approved Indication

Treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

What This Means For Patients

DAYBUE is not distributed through mainland China's retail pharmacy network. Uwell Cloud can help arrange a purchase channel for this medicine, sourced and dispatched from Hong Kong; clinical administration support in China can be coordinated on request. Availability and eligibility for any specific patient are determined by clinical review.

Regulatory Approvals

• FDA — Approved (2023) — Sources
• EMA — Approved (2026) — Sources

Pivotal Clinical Evidence

• LAVENDER — Neul et al., Nat Med 2023 (NCT04181723) — Sources

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/