Trofinetide
Trofinetide is a prescription medication approved for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. It is the first and only FDA-approved drug for this rare genetic neurological disorder. The exact mechanism by which trofinetide produces therapeutic effects is not fully understood, though it is believed to have neuroprotective and anti-inflammatory properties.
Trofinetide is an FDA-approved treatment for Rett syndrome that works through unknown mechanisms to improve symptoms in adults and children aged 2 and older.
Key facts at a glance
- ATC
- N07XX24
- RxNorm
- 2632852
- Generic
- Trofinetide
- Class
- atc-n07
- Brands
- Daybue
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- solution
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Trofinetide is indicated for the treatment of Rett syndrome, a rare genetic neurological disorder that primarily affects females and causes severe cognitive, motor, and communication impairments. It is approved for use in adults and pediatric patients 2 years of age and older.
The exact mechanism of action is unknown. Trofinetide is a synthetic analog of glycine-proline-glutamate (GPE), a naturally occurring peptide. Research suggests it may work through neurotrophic and anti-inflammatory pathways, potentially reducing neuroinflammation and supporting synaptic function in the brain.
Trofinetide is an oral solution taken twice daily. Common side effects may include diarrhea and vomiting. It should not be used in patients with known hypersensitivity to the drug. Patients and caregivers should report any signs of infection, as trofinetide may affect the immune system. Regular monitoring by a healthcare provider experienced in treating Rett syndrome is recommended.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy