Trofinetide

Overview

Trofinetide, sold under the brand name Daybue among others, is a medication used for the treatment of Rett syndrome. It is taken by mouth.[1] WP

The most common adverse reactions include diarrhea and vomiting.[2] WP

Trofinetide was approved for medical use in the United States in March 2023. The US Food and Drug Administration considers it to be a first-in-class medication.[1–5] WP

Uses, dosage and effects

Medical uses

Trofinetide is indicated for the treatment of Rett syndrome in people two years of age and older.[1][6] WP

Rett syndrome is a rare, genetic neurological and developmental disorder that affects the way the brain develops. People with Rett syndrome experience a progressive loss of motor skills and language. Most babies with Rett syndrome seem to develop as expected for the first six months of life. These babies then lose skills they previously had attained at approximately six to 18 months of age — such as the ability to crawl, walk, communicate, or use their hands. The hallmark of Rett syndrome is near constant repetitive hand movements, such as rubbing or clapping. Rett syndrome leads to severe impairments affecting nearly every aspect of life, including the ability to speak, walk, eat, and breathe.[2] WP

History

History

Trofinetide was developed by Neuren Pharmaceuticals that acts as an analogue of the neuropeptide (1-3) IGF-1, which is a simple tripeptide with sequence Gly-Pro-Glu obtained by enzymatic cleavage of the growth factor IGF-1 within the brain. Trofinetide has anti-inflammatory properties and was originally developed as a potential treatment for stroke, but has subsequently been developed for other applications and is approved by the FDA as an oral solution. It has successfully completed phase III clinical trial against Rett syndrome. Trofinetide has also had a successful phase II trial against Fragile X syndrome. It is manufactured by Acadia Pharmaceuticals.[1][7–13] WP

The US Food and Drug Administration (FDA) evaluated the efficacy and safety of trofinetide based on a randomized, double-blind, placebo-controlled, 12-week study (Study 1; NCT04181723) of participants with Rett syndrome 5 to 20 years of age. Participants were randomized to receive trofinetide (N=93) or matching placebo (N=94) for 12 weeks. The dose of trofinetide was based on participant weight to achieve similar exposure in all participants. The FDA granted the application for trofinetide priority review, orphan drug, and fast track designations.[2] WP

Legal status

Legal status

Trofinetide (Daybue) was approved for medical use in the United States in March 2023 and again in powder form (Daybue Stix) in December 2025.[2][14] WP

Daybu was authoried for medical use in the European Union in August 2026.[15][16] WP

Names

Trofinetide is sold under the brand names Daybue, Daybue Stix, and Daybu.[1][2][15] WP

Chemistry

Formula

C13H21N3O6 PC

CAS number

853400-76-7 PC

Molar mass (g/mol)

315.32 PC

IUPAC name (computed by PubChem)

(2S)-2-[[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino]pentanedioic acid PC

SMILES

C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)O PC

InChIKey

BUSXWGRAOZQTEY-SDBXPKJASA-N PC

PubChem CID

11318905

Harm reduction information

No harm reduction data is on file for this compound yet. That means no dose, timing or combination data from PsychonautWiki or TripSit. Please don't guess a dose from similar compounds. If you have taken it and feel unwell, contact emergency services or a poison control center.

User reports

Pop culture

No references added yet.

Sources

1. Journal / agency Daybue- trofinetide solution; Daybue Stix- trofinetide for solution. 18 August 2026
2. Journal / agency FDA approves first treatment for Rett Syndrome. 13 March 2023
3. Journal / agency Keam SJ. Trofinetide: First Approval. Drugs. June 2023. doi:10.1007/s40265-023-01883-8 · PMID 37191913
4. Journal / agency Drug Trials Snapshots: Daybue. 12 March 2023
5. Journal / agency New Drug Therapy Approvals 2023. January 2024
6. Journal / agency Drug Approval Package: Daybue. 6 April 2023
7. Journal / agency Bickerdike MJ, Thomas GB, Batchelor DC, Sirimanne ES, Leong W, Lin H, Sieg F, Wen J, Brimble MA, Harris PW, Gluckman PD. NNZ-2566: a Gly-Pro-Glu analogue with neuroprotective efficacy in a rat model of acute focal stroke. Journal of the Neurological Sciences. March 2009. doi:10.1016/j.jns.2008.12.003 · PMID 19157421
8. Journal / agency Cartagena CM, Phillips KL, Williams GL, Konopko M, Tortella FC, Dave JR, Schmid KE. Mechanism of action for NNZ-2566 anti-inflammatory effects following PBBI involves upregulation of immunomodulator ATF3. Neuromolecular Medicine. September 2013. doi:10.1007/s12017-013-8236-z · PMID 23765588
9. Other Positive top-line results from pivotal Phase 3 trial in Rett syndrome. 7 December 2021
10. Journal / agency Deacon RM, Glass L, Snape M, Hurley MJ, Altimiras FJ, Biekofsky RR, Cogram P. NNZ-2566, a novel analog of (1-3) IGF-1, as a potential therapeutic agent for fragile X syndrome. Neuromolecular Medicine. March 2015. doi:10.1007/s12017-015-8341-2 · PMID 25613838
11. Other Study Details - Rett Syndrome Study
12. Other Neuren's Tofinetide Successful in Phase 2 Clinical Trial in Fragile X. 7 December 2015
13. Other Acadia Pharmaceuticals Announces U.S. FDA Approval of Daybue (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older. 11 March 2023
14. Other Acadia Pharmaceuticals Announces FDA Approval of Daybue Stix (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome. 12 December 2025
15. Journal / agency Daybu EPAR. 27 February 2026
16. Other Daybu PI. 21 August 2026

Text and values on this page are quoted from Wikipedia (CC BY-SA 4.0), PubChem (public domain). Each value links to the exact revision it came from.