Description
Daybue (trofinetide) is a synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 (IGF-1), approved for the treatment of Rett syndrome in pediatric and adult patients aged two years and older. Rett syndrome is a rare neurodevelopmental disorder primarily affecting females and is characterized by a period of normal development followed by a loss of acquired skills, including communication and motor abilities. Trofinetide is believed to exert its effects by reducing neuroinflammation and supporting synaptic function. It is the first FDA-approved treatment specifically indicated for Rett syndrome.
| Fact Table |
| Formula | C25H38N6O7 |
| License | US FDA (2023) |
| Bioavailability | ~70% |
| Legal status | Rx-only (USA) |
| Chemical Name | (2S)-2-[[(2S)-2-[[(2S)-2-(2-methoxybenzylcarbamoyl) pyrrolidine-1-carbonyl]amino] propanoyl]amino]propanoic acid |
| Elimination half-life | 1.5 hours |
| Dosage (Strength) | 200 mg/mL oral solution |
| Pregnancy | Consult a doctor (no human data; animal studies show risk) |
| Brands | Daybue |
| Protein binding | <20% |
| PubChem CID | 160948 |
| MedlinePlus | a623030 |
| ChEBI | 86366 |
| ATC code | N07XX18 |
| DrugBank | DB05527 |
| KEGG | D12686 |
| Routes of administration | Oral (solution) |
Directions
Daybue is administered orally, typically twice daily, in the morning and evening. The recommended dose is based on body weight. For example, patients weighing =50 kg should receive 12 g twice daily. It is supplied as an oral solution and can be taken with or without food. The solution should be measured using the provided dosing device, and caregivers should follow specific instructions for administration. Dose adjustments may be necessary in patients with severe renal impairment.
Ingredients
Each mL of Daybue oral solution contains:
The formulation is free of preservatives and dyes.
Contraindications
Daybue is contraindicated in patients with a known hypersensitivity to trofinetide or any of the formulation's excipients. Hypersensitivity reactions may include rash, urticaria, or anaphylaxis.
Cautions
Diarrhea is a common and potentially dose-limiting side effect of Daybue; therefore, fluid status should be monitored, especially in pediatric patients. Discontinuation or dose reduction may be necessary in cases of severe diarrhea. Use in patients with severe renal impairment (eGFR <30 mL/min/1.73 m²) should be avoided. Clinical response and tolerability should be reassessed periodically, especially during dose titration or in patients with fluctuating weight.
Side Effects
Common side effects of Daybue include:
- Upper respiratory tract infections
Serious adverse effects may include severe diarrhea leading to dehydration and electrolyte imbalance. Monitoring of liver function tests and hydration status is recommended during treatment.
IMPORTANT NOTE:
The above information is intended to increase awareness of health information and does not suggest treatment or diagnosis. This information is not a substitute for individual medical attention and should not be construed to indicate that use of the drug is safe, appropriate, or effective for you. See your health care professional for medical advice and treatment.
Product Code : 14947
Prescription Required