Trofinetide
FDA approved · RxSynthetic IGF-1 tripeptide analog
Also known as: Daybue, Daybue Stix, NNZ-2566, DAYBUE, ACP-2566, trofinetida, trofinetidum, Glycyl-2-methyl-L-prolyl-L-glutamic acid
Strong, human-grade evidence
- Regulatory standing. FDA-approved prescription medicine
- Human trials. Backed by controlled human studies
- Registered human trials. 14 registered studies on ClinicalTrials.gov, most advanced Phase 3
- 2 cited sources. Includes primary / regulatory sources
Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.
What it is
Trofinetide is a synthetic analog of glypromate (glycine-proline-glutamate), a tripeptide fragment naturally cleaved from insulin-like growth factor 1 (IGF-1) in the brain. It is thought to act by reducing neuroinflammation and supporting synaptic function, though its exact mechanism is not fully understood. It is sold under the brand names Daybue and Daybue Stix.
What the evidence shows
Trofinetide is FDA approved for the treatment of Rett syndrome in adults and children aged 2 years and older, based on Phase 3 trial results showing modest improvements on symptom rating scales. It has been studied in 14 registered human trials, and evidence for uses beyond Rett syndrome, such as other neurodevelopmental conditions, remains limited.
Key facts
Registered human trials
Live from ClinicalTrials.gov, refreshed daily.
Safety Study of NNZ-2566 in Healthy Subjects, Following Oral Administration
Open-Label Extension Study of Trofinetide for Rett Syndrome
Cognitive Function in Rett Syndrome During Trofinetide Treatment
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
Published literature
Live from Europe PMC. This is how much has been published, not how much is proven in humans.
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
An Update on Psychopharmacological Treatment of Autism Spectrum Disorder.
Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.
A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome.
Sources
- PubChem compound record (CID 11318905)
- FDA drug label on DailyMed
- Registered trials on ClinicalTrials.gov
- Published papers on Europe PMC
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