Trofinetide

The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown.

• DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

• None. None.contraindicated

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.

Diarrhea: Most patients experience diarrhea during treatment with DAYBUE. Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should start antidiarrheal treatment, increase oral fluids, and notify their healthcare provider. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if severe diarrhea occurs or if dehydration is suspected. ( 2.4 , 5.1 ) Weight Loss: Weight loss may occur in patients treated with DAYBUE or DAYBUE STIX. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if significant weight loss occurs. ( 2.4 , 5.2 ) Vomiting: Aspiration and aspiration pneumonia have occurred after vomiting in patients treated with DAYBUE. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if vomiting is severe or occurs despite medical management. ( 2.5 , 5.3 ) 5.1 Diarrhea In Study 1 [see Clinical Studies (14) ] and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was of mild or moderate severity in 96% of cases. In Study 1, antidiarrheal medication was used in 51% of patients treated with DAYBUE. Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed.

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 Age and Older The safety of DAYBUE was evaluated in a randomized, double-blind, placebo-controlled, 12-week study of patients with Rett syndrome (Study 1) [see Clinical Studies (14) ] .

Severe renal impairment: DAYBUE and DAYBUE STIX are not recommended. ( 8.6 ) 8.1 Pregnancy Risk Summary There are no adequate data on the developmental risks associated with the use of DAYBUE or DAYBUE STIX in pregnant women. No adverse developmental effects were observed following oral administration of trofinetide to pregnant animals at doses associated with plasma exposures below those used clinically [see Animal Data ] . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data Oral administration of trofinetide (0, 150, 450, or 1000 mg/kg twice daily; 0, 300, 900, or 2000 mg/kg/day) to pregnant rats during the period of organogenesis resulted in no adverse effects on embryofetal development. At the highest dose tested, plasma exposure (AUC) was less than that in humans at the maximum recommended human dose (MRHD) of 12,000 mg twice daily (24,000 mg/day). Oral administration of trofinetide (0, 75, 150, or 300 mg/kg twice daily; 0, 150, 300, or 600 mg/kg/day) to pregnant rabbits during the period of organogenesis resulted in no adverse effects on embryofetal development.

Metabolism

DAYBUE STIX for oral solution demonstrated comparable bioavailability to DAYBUE oral solution in adult healthy subjects under fasted state. Trofinetide exhibits linear kinetics with no time- or dose-dependent effect on pharmacokinetic parameters.

• Mar 10, 2023NDANDA217026Acadia Pharms Inc
• Dec 11, 2025NDANDA219884Acadia Pharms Inc

1. 1Diarrhoea3,29455%
2. 2Underdose1,84331%
3. 3Vomiting1,06618%
4. 4Product Dose Omission Issue85914%
5. 5Seizure75313%
6. 6Constipation61310%
7. 7Product Use Complaint5799.7%
8. 8Weight Decreased5158.6%
9. 9Drug Ineffective4327.2%
10. 10Intentional Dose Omission4267.1%
11. 11Decreased Appetite3896.5%
12. 12Nasopharyngitis3576.0%
13. 13Flatulence3375.6%
14. 14Faeces Soft3275.5%
15. 15Inappropriate Schedule Of Product Administration3265.4%

The 10 most recently updated of 13 ClinicalTrials.gov registrations naming Trofinetide as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Cognitive Function in Rett Syndrome During Trofinetide Treatment
  Recruiting · Observational · 20 enrolled · University of Minnesota
  NCT06705816updated 2025-06-12
• Open-Label Extension Study of Trofinetide for Rett Syndrome
  Terminated · Phase 3 · Interventional · 77 enrolled · ACADIA Pharmaceuticals Inc.
  NCT04776746updated 2024-09-27
• An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
  Terminated · Phase 2 · Phase 3 · Interventional · 15 enrolled · ACADIA Pharmaceuticals Inc.
  NCT04988867updated 2024-09-24
• Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
  Completed · Phase 3 · Interventional · 154 enrolled · ACADIA Pharmaceuticals Inc.
  NCT04279314updated 2024-04-11
• Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
  Completed · Phase 3 · Interventional · 187 enrolled · ACADIA Pharmaceuticals Inc.
  NCT04181723updated 2024-04-08
• A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
  Completed · Phase 2 · Interventional · 82 enrolled · Neuren Pharmaceuticals Limited
  NCT02715115updated 2020-08-14
• Study of NNZ-2566 in Patients With Traumatic Brain Injury Under EFIC
  Completed · Phase 2 · Interventional · 261 enrolled · Neuren Pharmaceuticals Limited
  NCT01366820updated 2018-02-05
• A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
  Completed · Phase 2 · Interventional · 72 enrolled · Neuren Pharmaceuticals Limited
  NCT01894958updated 2018-02-05
• A Safety Study of NNZ-2566 in Patients With Rett Syndrome
  Completed · Phase 2 · Interventional · 67 enrolled · Neuren Pharmaceuticals Limited
  NCT01703533updated 2018-02-05
• Study of NNZ-2566 in Patients With Traumatic Brain Injury
  Completed · Phase 2 · Interventional · 261 enrolled · Neuren Pharmaceuticals Limited
  NCT00805818updated 2018-02-05

Frequently asked questions

How does Trofinetide work?

The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown.

What is Trofinetide used for?

According to FDA labeling, Trofinetide carries indications including: DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Trofinetide?

Trofinetide is classified as Other nervous system drugs, Cytochrome P450 3A4 Inhibitors, Organic Anion Transporting Polypeptide 1B1 Inhibitors, Organic Anion Transporting Polypeptide 1B3 Inhibitors, Unknown Cellular or Molecular Interaction.

What are the brand names for Trofinetide?

Trofinetide is marketed under brand names including Daybue.

What are the contraindications for Trofinetide?

Trofinetide labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.