Trofinetide
DAYBUE
What it isSynthetic glycine-proline-glutamate analog derived from IGF-1; Rett syndrome.
Why this oneIt was the first drug labeled specifically for Rett syndrome, with benefit measured across core symptoms.
What limits itDiarrhea, vomiting, and weight loss frequently end treatment; the required oral-solution volume adds burden.
FDA-approved for
- Rett syndrome (2023)
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Rett syndrome | Patients ≥2 yr (weight-based) | 9–<12 kg: 5,000 mg; 12–<20 kg: 6,000 mg; 20–<35 kg: 8,000 mg; 35–<50 kg: 10,000 mg; ≥50 kg: 12,000 mg | per weight band BID | 12,000 mg BID (≥50 kg) |
Renal Mild (eGFR 60–89): no adjustment. Moderate (eGFR 30–59): reduced per-weight dose. Severe (<30): not recommended
Hepatic Undefined
Instructions
- Twice daily, morning and evening, with or without food
- Oral or via gastrostomy (G) tube; GJ tubes must use the G-port
- Stop laxatives before starting
- STIX: dissolve powder in 15–120 mL cold/room-temp water or water-based beverage; administer immediately
- Discard opened DAYBUE oral solution after 14 days
- If vomiting after a dose, do not repeat — take the next scheduled dose
- Missed dose: take next dose as scheduled; do not double
Cautions
- Diarrhea (very common) — dehydration risk; interrupt/reduce/discontinue if severe
- Weight loss — monitor weight
- Vomiting — aspiration and aspiration pneumonia have occurred
Adverse reactions
- Diarrhea
- Vomiting
- Fever
- Seizure
- Anxiety
- Decreased appetite
- Fatigue
- Nasopharyngitis
Pregnancy No adequate human data; no adverse developmental effects in animals at exposures below clinical
Lactation No data; weigh benefits of breastfeeding against maternal need
NO BOXED WARNINGS
Principal riskDiarrhea in more than four-fifths of patients
Cost, est. cash$300–$5,000/month
Generic entry2040 est.
Classes and tags
Clinical profile
Legacy pregnancy categoryNot assigned — PLLR-era drug
Defining liabilityGenerally tolerated at therapeutic doses; clinically important risks are agent-specific and increase with interactions or organ impairment.
Mechanism of action: Trofinetide ballicule
