Trofinetide

Trofinetide is a prescription medication used for the treatment of Rett Syndrome.

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

Common side effects of Trofinetide include:

• Diarrhea, 
• Vomiting,
• Fever, 
• Seizure, 
• Anxiety, 
• Decreased appetite, 
• Tiredness, and the common cold.

Serious side effects of Trofinetide include:

• Diarrhea, 
• Vomiting,
• Weight loss.

Rare side effects of Trofinetide include:

• None 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

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.

Adult and pediatric dosage

Oral solution

• 200 mg/mL (450 mL/bottle)

Rett Syndrome

Adult dosage

• 35 kg to below 50 kg: 10,000 mg (50 mL) orally two times a day
• Above 50 kg: 12,000 mg (60 mL) orally two times a day

Pediatric dosage

• 9 kg to below 12 kg: 5,000 mg (25 mL) orally two times a day
• 12 kg to below 20 kg: 6,000 mg (30 mL) orally two times a day
• 20 kg to below 35 kg: 8,000 mg (40 mL) orally two times a day
• 35 kg to below 50 kg: 10,000 mg (50 mL) orally two times a day
• Above 50 kg: 12,000 mg (60 mL) orally two times a day

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Trofinetide has severe interactions with no other drugs.
• Trofinetide has serious interactions with at least 40 other drugs.
• Trofinetide has moderate interactions with at least 29 other drugs.
• Trofinetide has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Trofinetide?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Trofinetide?”

Cautions

• Diarrhea
  • Diarrhea is expected in most patients and ranges in severity.
  • Advise patients to stop laxatives before starting.
  • If diarrhea occurs, have the patient notify the healthcare provider.
  • Consider starting antidiarrheal treatment, monitor hydration status, and increase oral fluids, if needed
  • Interrupt, reduce the dose, or discontinue if severe diarrhea occurs or if dehydration is suspected.
• Weight loss
  • Weight loss may occur.
  • Monitor weight and interrupt, reduce dose, or discontinue if significant weight loss occurs.
• Drug interaction overview
  • Inhibitor of CYP3A4 (weak), OATP1B1, and OATP1B3
  • Sensitive CYP3A4 substrates
    • Closely monitor.
      • Coadministration may increase plasma concentrations of orally administered CYP3A4 sensitive substrates.
      • Monitor CYP3A4 substrates for which a small change in substrate plasma concentration may lead to serious toxicities.
  • Sensitive OATP1B1 or OATP13 substrates
    • Avoid coadministration.
      • Trofinetide inhibits OATP1B1 and OATP1B3 in vitro.
      • Avoid substrates for which a small change in substrate plasma concentration may lead to serious toxicities.

Pregnancy and Lactation

• There are no adequate data on developmental risks associated with use in pregnant females.
• Lactation
  • Data are not available regarding the presence of trofinetide or its metabolites in human milk, its effects on breastfed infants, or its effects on milk production.