Trofinetide

Trofinetide is used to treat Rett syndrome in adults and children 2 years of age and older. The exact way this medicine works to treat the symptoms of Rett syndrome is not known.

• Trofinetide is available under the following different brand names: Daybue.

Common side effects of Trofinetide include:

• Diarrhea
• Vomiting
• Fever
• Seizures
• Anxiety
• Loss of appetite
• Tiredness
• Infection in the nose or throat, sore throat, or common cold symptoms

Serious side effects of Trofinetide include:

• Diarrhea. Diarrhea is common with Trofinetide. Call your health care provider right away if you have diarrhea or if you have more than two bowel movements in one day. If you are given medicine to prevent or treat diarrhea, take it exactly as your health care provider tells you. Call your health care provider right away if you have severe diarrhea, diarrhea that does not go away, or diarrhea with weakness, dizziness, or fever.
• Weight loss. Trofinetide can cause weight loss. Tell your health care provider if you notice you are losing weight at any time during treatment with Trofinetide.
• Vomiting. Trofinetide can cause vomiting. Sometimes vomit can get into your lungs (aspiration), which could cause an infection (aspiration pneumonia). Tell your health care provider if you have severe vomiting or if vomiting happens often.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Dosage Forms & Strengths

• Oral solution
• Powder for oral solution

Rett Syndrome

Adult Dose

• 50 kg or more: 12,000 mg twice daily

Pediatric Dose

Recommended dose in children 2 years and older is based on weight.

• 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

Trofinetide may alter the blood levels of other medicines. Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Trofinetide is right for you. Tell your health care provider about all medicines that you take or have recently taken.

This may not be a complete list of medicines that can interact with Trofinetide. Always check with your health care provider.

Contraindications: None

Cautions

• Pregnancy: It is not known if Trofinetide will harm an unborn baby. Talk to your health care provider if you are pregnant or plan to become pregnant.
• Lactation: It is not known if Trofinetide passes into breast milk. Talk to your health care provider about the best way to feed your baby while taking Trofinetide.