Trofinetide

What is Trofinetide?

• Trofinetide (Daybue), is an analog of glycine-proline-glutamate (GPE), a protein that occurs naturally in the brain.

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Trofinetide

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Trofinetide structure

What are the uses of this medicine?

• Trofinetide (Daybue) is a prescription medicine used to treat Rett syndrome in adults and children 2 years of age and older.

How does this medicine work?

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

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
• Your doctor may need to change the doses of your medications or monitor you carefully for side effects.

Be sure to mention any of the following:

• laxatives
• Orally administered CYP3A4 sensitive substrates
• OATP1B1 and OATP1B3 substrates

Is this medicine FDA approved?

• Trofinetide was approved for medical use in the United States in March 2023.

How should this medicine be used?

Recommended dosage: Administer Daybue orally twice daily, in the morning and evening, according to patient weight as shown:

• 9 kg to less than 12 kg: 5,000 mg twice daily(25 mL twice daily)
• 12 kg to less than 20 kg: 6,000 mg twice daily(30 mL twice daily)
• 20 kg to less than 35 kg: 8,000 mg twice daily(40 mL twice daily)
• 35 kg to less than 50 kg: 10,000 mg twice daily (50 mL twice daily)
• 50 kg or more: 12,000 mg twice daily (60 mL twice daily)

Administration:

• Take Daybue exactly as your healthcare provider tells you to take it.
• If you take laxatives, stop taking them before starting treatment with Daybue.
• Your healthcare provider may change your dose or stop treatment with Daybue if needed.
• Your pharmacist should provide an oral syringe or dosing cup that is needed to measure your prescribed dose. Do not use a household measuring cup.
• Daybue may be taken by mouth or given through a gastrostomy (G) tube. If Daybue is given through a gastrojejunal (GJ) tube, the G-port must be used.
• Daybue may be taken with or without food.
• Throw away any unused Daybue after 14 days of first opening the bottle.
• If you vomit after taking a dose of Daybue, do not take another dose to make up for that dose. Wait and take the next dose at your usual time.
• If you miss a dose of Daybue, skip that dose and take your next dose at your usual time. Do not take 2 doses to make up the missed dose.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Oral solution: 200 mg/mL

This medicine is available in fallowing brand namesː

• Daybue

What side effects can this medication cause?

The most common side effects of this medicine include:

• Diarrhea
• Vomiting
• Fever
• Seizure
• Anxiety
• Decreased appetite
• Fatigue
• Nasopharyngitis

Less common, but serious side effects may include:

• Diarrhea
• Weight loss

What special precautions should I follow?

• Most patients experience diarrhea during treatment with Daybue. Advise patients to stop laxatives before starting Daybue. If diarrhea occurs, patients should start antidiarrheal treatment, increase oral fluids, and notify their healthcare provider. Interrupt, reduce dose, or discontinue Daybue if severe diarrhea occurs or if dehydration is suspected.
• Weight loss may occur in patients treated with Daybue. Monitor weight and interrupt, reduce dose, or discontinue Daybue if significant weight loss occurs.
• Advise the caregiver or patient that Daybue can cause vomiting and if vomiting occurs after Daybue administration, do not take an additional dose, but continue with the next scheduled dose.
• Advise the caregiver or patient that Daybue may be given orally or via gastrostomy (G) tube; doses administered via gastrojejunal (GJ) tubes must be administered through the G-port. Daybue may be taken with or without food.
• Instruct the caregiver or patient to obtain a calibrated measuring device, such as an oral syringe or oral dosing cup, from the pharmacy to measure and deliver the prescribed dose accurately. A household measuring cup is not an adequate measuring device.
• Instruct the caregiver or patient to discard any unused Daybue after 14 days of first opening the bottle.
• It is not known if Daybue passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby while taking Daybue.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no adequate data on the developmental risks associated with the use of Daybue in pregnant women.
• It is not known if Daybue will harm your unborn baby.

Can this medicine be used in children?

• The safety and effectiveness of Daybue for the treatment of Rett syndrome have been established in pediatric patients aged 2 years and older.
• Safety and effectiveness in pediatric patients less than 2 years of age have not been established.

What are the active and inactive ingredients in this medicine?

• Active ingredient: trofinetide
• Inactive ingredients: FD&C Red No. 40, maltitol, methylparaben sodium, propylparaben sodium, purified water, strawberry flavor, and sucralose.

Who manufactures and distributes this medicine?

• Marketed by Acadia Pharmaceuticals Inc., San Diego, CA, USA
• Daybue is a trademark of Acadia Pharmaceuticals Inc.

What should I know about storage and disposal of this medication?

• Store Daybue in the refrigerator between 36°F to 46°F (2°C to 8°C). Do not freeze.
• Keep Daybue in an upright position.
• Keep the child-resistant cap tightly closed.

• Dailymed label info on Trofinetide
• FDA Trofinetide

Trofinetide Resources
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