Trofinetide
Also referenced as: Daybue, NNZ-2566, Trofinetide predecessor programs
A synthetic analogue of an IGF-1-derived tripeptide, developed as a treatment for Rett syndrome.
- Evidence grade
- BModerate Human Evidence
Moderate Human Evidence
- Regulatory status
- FDA Approved for Specific Indication
FDA-approved Daybue for Rett syndrome in patients aged 2 years and older; not approved for general cognition or anti-aging.
- Human data
- Yes
Registered clinical trials exist
- Last reviewed
- October 27, 2026
Overview
A synthetic analogue of an IGF-1-derived tripeptide, developed as a treatment for Rett syndrome.
FDA-approved Daybue for Rett syndrome in patients aged 2 years and older; not approved for general cognition or anti-aging.
Development codes and predecessor-program headings are not separate medicines. Outcomes in Rett syndrome cannot be generalized to healthy readers.
Why It's Being Studied
- Rett syndrome
- Neurodevelopmental symptoms
Mechanism of Action
Its therapeutic mechanism is not established; proposed neuroinflammatory and synaptic effects are not proof of how the medicine produces clinical responses.
Its therapeutic mechanism is not established; proposed neuroinflammatory and synaptic effects are not proof of how the medicine produces clinical responses. Development codes and predecessor-program headings are not separate medicines. Outcomes in Rett syndrome cannot be generalized to healthy readers.
From molecular target to clinical evidence
Tap each stage to see what happens.
Background: Clinical Peptide Medicines
Clinical peptide medicines include endocrine treatments, antimicrobial drugs, receptor blockers and targeted diagnostic or cancer agents. They do not share one mechanism or one set of indications.
An approval belongs to a specific medicine and indication. A diagnostic tracer, an antibiotic and a hormone are not interchangeable wellness products; read each profile's own mechanism and prescribing source.
Research Areas & Routes Studied
- Rett syndrome
- Neurodevelopmental symptoms
- Oral approved medicine
Evidence Summary
The randomized LAVENDER study supports symptom-scale improvement in Rett syndrome. FDA approval is indication-specific, not evidence of general cognitive enhancement.
Higher tiers carry more weight. Filled tiers have evidence for Trofinetide; tap to read it.
- The randomized LAVENDER study supports symptom-scale improvement in Rett syndrome. FDA approval is indication-specific, not evidence of general cognitive enhancement.
2 cited sources.
- Randomised controlled trial1
- Prescribing label1
Human clinical evidence
- The randomized LAVENDER study supports symptom-scale improvement in Rett syndrome. FDA approval is indication-specific, not evidence of general cognitive enhancement.
Reported Adverse Events in Research
- Diarrhea, vomiting and weight loss are important risks; vomiting may create aspiration risk.
Limitations of Current Evidence
- Development codes and predecessor-program headings are not separate medicines. Outcomes in Rett syndrome cannot be generalized to healthy readers.
- Findings apply to the product, route and population studied; they do not establish benefits or safety for unrelated wellness uses or research-vial products.
How to Read This Research
- Is this a single molecule or a family of medicines?
- Is the use diagnostic, therapeutic or investigational?
- Does the quoted outcome match the exact product, formulation and population?
- Use the compound-specific mechanism above; this diagram describes drug development, not a shared biological pathway.
- Historical approval, present availability and off-label use are distinct questions.
- For radiopharmaceuticals and hospital medicines, specialist handling is part of the treatment context.
- Indication
- The condition and population for which a medicine is approved.
- Antagonist
- A compound that blocks activity at a receptor.
- Radiopharmaceutical
- A medicine containing a radioactive isotope for imaging or treatment.
Compare With Other Compounds
| Trofinetide | Ziconotide | |
|---|---|---|
| Evidence grade | B — Moderate Human Evidence | A — Strong Clinical Evidence |
| Regulatory status | FDA Approved for Specific Indication | FDA Approved for Specific Indication |
| Human data | Yes | Yes |
| Clinical trials | Registered trials exist | Registered trials exist |
| Research routes | Oral approved medicine | Intrathecal approved medicine |
| Cited sources | 2 | 1 |
Frequently Asked Questions
Research References
- Randomised controlled trial
Trofinetide — randomised controlled trial
- Prescribing label
Daybue prescribing information