trofinetide
Daybu · ATC code N07XX24
Also known as: Trofinetid
- Key facts
- Active substance
- trofinetide
- ATC code
- N07XX24
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Daybu 200 mg/mL oral solution
- Marketing authorisation holder
- Acadia Pharmaceuticals B.V.
- Information as of
- 2026-10-28
Key facts
Trofinetide (Daybu) is an oral solution used to treat neurobehavioural symptoms of Rett syndrome in adults and children aged 5 years and older.
What it is used for
- Treatment of neurobehavioural symptoms of Rett syndrome in adults and paediatric patients aged 5 years and older.
Dosing
- Oral solution twice daily (morning and evening) by weight: 5 g (9 kg to less than 12 kg), 6 g (12 kg to less than 20 kg), 8 g (20 kg to less than 35 kg), 10 g (35 kg to less than 50 kg) and 12 g (50 kg or more), each twice daily.
- Moderate renal impairment (eGFR 30 to 59): reduced doses of 2.5 g to 6 g twice daily depending on weight; severe renal impairment (eGFR 15 to 29): not recommended.
- No dose adjustment in hepatic impairment or mild renal impairment.
- Consider interruption, dose reduction or discontinuation for severe diarrhoea, vomiting or significant weight loss; no extra dose after vomiting.
Contraindications
- Hypersensitivity to the active substance or any excipient.
Important warnings
- Diarrhoea: stop laxatives before starting; treat with antidiarrhoeals and monitor hydration.
- Vomiting: patients with Rett syndrome are at risk of aspiration; aspiration and aspiration pneumonia have been reported.
- Weight loss: monitor body weight.
- Close monitoring with sensitive CYP3A4 and/or P-gp substrates (e.g. cyclosporine, digoxin).
Side effects
- Most common (12-week study): diarrhoea (80.6%) and vomiting (26.9%).
- 43.3% of patients discontinued because of adverse reactions, mostly diarrhoea (25.8%), vomiting (6.7%), seizures (2.8%), weight loss (2.2%) and decreased appetite (2.2%).
- Others: irritability, seizures.
Interactions
- Weak inhibitor of CYP3A4 and P-gp: levels of their substrates (e.g. loperamide, cyclosporine, digoxin) may increase.
- Possible inhibition of MATE1/MATE2-K (e.g. metformin).
Pregnancy and breastfeeding
- Not recommended during pregnancy or in women of childbearing potential not using contraception; effective contraception during treatment.
Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
Common questions – ask Medelix
For patients
Package leaflet, verbatim
This product has no separate US patient information.
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- DaybuAcadia Pharmaceuticals B.V. · EMEA/H/C/006482
Source: European Medicines Agency (EMA), product information
Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.
Other substances in the same group
Another question about this substance?
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Sources
- European Medicines Agency. Daybu – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/daybu-epar-product-information_en.pdf
Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.