Trofinetide: uses, dosing, side effects & brands
Trofinetide is a medicine sold in the U.S. under one brand, for rett syndrome. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
Key facts
- Treats
- Rett Syndrome
- Available as
- Solution
- Sold as
- Daybue
- Prescription?
- Prescription only
- Generic available?
- Not in our catalog
How trofinetide is dosed
From the FDA label for Daybue (application NDA217026). Other trofinetide products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
Recommended dosage is twice daily, morning and evening, according to patient weight. DAYBUE or DAYBUE STIX can be given with or without food. ( 2.1 ) Patient Weight Recommended Dosage 9 kg to less than 12 kg 5,000 mg twice daily 12 kg to less than 20 kg 6,000 mg twice daily 20 kg to less than 35 kg 8,000 mg twice daily 35 kg to less than 50 kg 10,000 mg twice daily 50 kg or more 12,000 mg twice daily Can be given orally or via gastrostomy (G) tube; doses administered via gastrojejunal (GJ) tubes must be administered through the G-port. ( 2.1 ) See Full Prescribing Information for instruction on dissolving DAYBUE STIX for oral solution powder. ( 2.3 ) See Full Prescribing Information for dosage recommendations in patients with renal impairment. ( 2.6 , 8.6 ) 2.1 Recommended Dosage The recommended dosage of DAYBUE or DAYBUE STIX is based on patient weight as shown in Table 1 . Administer DAYBUE or DAYBUE STIX orally or via gastrostomy (G) tube twice daily, in the morning and evening, with or without food. Doses administered via gastrojejunal (GJ) tubes must be administered through the G-port. Table 1 Recommended Dosage of DAYBUE or DAYBUE STIX in Patients 2 Years of Age and Older Patient Weight Recommended Dosage 9 kg to less than 12 kg 5,000 mg twice daily 12 kg to less than 20 kg 6,000 mg twice daily 20 kg to less than 35 kg 8,000 mg twice daily 35 kg to less than 50 kg 10,000…
Trofinetide side effects
The following clinically significant adverse reactions are described elsewhere in labeling: Diarrhea [see Warnings and Precautions (5.1) ] Weight Loss [see Warnings and Precautions (5.2) ] Vomiting [see Warnings and Precautions (5.3) ] The most common adverse reactions (that occurred in at least 10% of DAYBUE-treated patients and at least 2% greater than in placebo) were diarrhea and vomiting. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Acadia Pharmaceuticals Inc. at 1-844-422-2342 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In controlled and uncontrolled trials in patients with Rett syndrome, 260 patients ages 2 to 40 years were treated with DAYBUE, including 112 patients treated for more than 6 months, 92 patients treated for more than 1 year, and 44 patients treated for more than 2 years. The safety of DAYBUE STIX has been established from an adequate, well-controlled study, and open-label studies of DAYBUE [see Clinical Studies (14) ] . Below is a display of the adverse reactions of DAYBUE in these studies. Adult and Pediatric Patients with Rett Syndrome 5 Years of…
Who shouldn’t take trofinetide
None. None. ( 4 )
Trofinetide drug interactions
Orally administered CYP3A and/or P-gp sensitive substrates for which a small change in substrate plasma concentration may lead to serious adverse reactions: closely monitor for adverse reactions with concomitant use. ( 7.1 ) 7.1 Effect of DAYBUE and DAYBUE STIX on Other Drugs CYP3A and/or P-gp Substrates Closely monitor patients when DAYBUE or DAYBUE STIX is administered concomitantly with sensitive CYP3A and/or P-gp substrates where minimal increases in the plasma concentration of these substrates may lead to serious adverse reactions. Trofinetide, a weak inhibitor of CYP3A and an inhibitor of P-gp, increased the plasma concentrations of CYP3A and/or P-gp substrates [see Clinical Pharmacology (12.3) ] , which may increase the risk of adverse reactions associated with these substrates.
Every trofinetide product we track (1)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
Only one: Daybue.
What people report to the FDA about trofinetide
The FDA Adverse Event Reporting System (FAERS) collects reports from patients and clinicians. It holds 21 reports naming trofinetide, and the FDA flagged 76% of those reports as serious. The effects reported most often — leaving out reports about overdose, misuse or the condition being treated, which dominate the raw list for common medicines:
- diarrhoea4 reports
- pneumonia4 reports
- seizure4 reports
- status epilepticus4 reports
- constipation3 reports
- fall2 reports
- infection2 reports
- nasopharyngitis2 reports
Read these as a signal, not a rate. A report does not mean trofinetide caused the effect — anyone can file one, and many describe people taking several medicines for several conditions. Crucially there is no denominator: FAERS does not record how many people took the drug, so these counts cannot be turned into “X% of patients” — a bigger number often just means a more widely used or more talked-about drug. Duplicates exist, and publicity drives reporting. For what is actually established, read the FDA label section above.
Source: openFDA drug/event (FAERS), retrieved July 26, 2026.
Related calculators
Frequently asked questions
What is trofinetide?
DAYBUE oral solution and DAYBUE STIX for oral solution contain the active moiety trofinetide.
Can you take trofinetide with other medicines?
It depends on the medicine. We check it against the FDA labels rather than guessing: our interaction checker searches each drug's own label for the other and quotes what it says, naming the section it came from. Run trofinetide against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What forms does trofinetide come in?
Across the brands we track, trofinetide is currently marketed as solution, per the FDA's National Drug Code Directory. Each form is dosed differently — follow the label for the exact product you were prescribed.
Is there a generic trofinetide?
We do not currently list a generic-labelled trofinetide product. That does not always mean none exists — it means none appears under a generic name in the FDA data we track. Ask your pharmacist.
Cite this page
- APA
- pharmaranks. (2026, July 24). Trofinetide: uses, dosing, side effects & brands. https://pharmaranks.com/drugs/trofinetide
- MLA
- “Trofinetide: uses, dosing, side effects & brands.” pharmaranks, 24 July 2026, https://pharmaranks.com/drugs/trofinetide.
We summarise public FDA and NIH sources — for a clinical claim, cite the primary source we link to as well.
Sources: FDA openFDA drug label, National Drug Code Directory, and Enforcement (recall) database. This page reproduces public FDA data and is not medical advice. Dosing is set by your prescriber.
Read the full FDA label for trofinetide on DailyMed (NIH) ↗.