Why is this medication prescribed?

Trofinetide is used to treat Rett syndrome (an inherited condition that causes severe developmental and nervous system problems) in adult and children 2 years of age or older. Trofinetide is an analog of glycine-proline-glutamate (GPE), a protein that occurs naturally in the brain. It is not known exactly how trofinetide works, but it may improve nerve function and structure.

How should this medicine be used?

Trofinetide is available as a liquid solution for oral use. It is often taken twice a day, either with or without meals, in the morning and the evening. Take trofinetide daily at approximately the same times. Pay close attention to the instructions on the label of your prescription and ask your pharmacist or doctor to explain anything you do not understand. Follow the directions on trofinetide exactly. Never take more or less of it, or take it more frequently than your doctor has instructed.

To measure and take your trofinetide solution dose precisely, use an oral syringe or dosing cup (a measuring device); do not use a regular spoon or cup. If your prescription does not come with an oral syringe or cup, ask your pharmacist for one.

Don’t take another dose of trofinetide if you throw up after taking it. The following day, stick to your usual dose schedule.

Your physician or pharmacist will provide instructions on how to administer trofinetide solution through a nasogastric (NG) tube if you have one.

Request a copy of the patient’s manufacturer’s information from your pharmacist or physician.

Other uses for this medicine

Ask your doctor or pharmacist for more information if you think this medicine could be recommended for something else.

What special precautions should I follow?

Regarding special precautions for Trofinetide, here are some general considerations:

• Medical History: Inform your healthcare provider about any medical conditions you have, especially neurological or psychiatric disorders, as well as any allergies or sensitivities.
• Medication Interactions: Discuss all medications, supplements, and herbal remedies you are taking with your healthcare provider to avoid potential interactions.
• Pregnancy and Breastfeeding: If you are pregnant, planning to become pregnant, or breastfeeding, discuss the risks and benefits of Trofinetide with your healthcare provider. It’s essential to weigh the potential benefits against any possible risks to the fetus or infant.
• Monitoring: Your healthcare provider may need to monitor your response to Trofinetide regularly, including assessing for any side effects or changes in symptoms.
• Safety Precautions: If Trofinetide is administered via injection, follow proper safety protocols and techniques to minimize the risk of infection or injury.
• Adherence to Dosage Instructions: Take Trofinetide exactly as prescribed by your healthcare provider. Do not adjust the dosage or frequency without consulting them first.
• Side Effects: Be aware of potential side effects of Trofinetide and report any adverse reactions to your healthcare provider promptly.
• Storage: Store Trofinetide according to the instructions provided by the manufacturer or pharmacist, typically at room temperature away from moisture and heat.

Always consult your healthcare provider for specific precautions and guidance tailored to your individual circumstances before starting or discontinuing any medication.

What special dietary instructions should I follow?

• Trofinetide does not typically require specific dietary restrictions. However, it’s always a good idea to maintain a balanced and healthy diet as advised by your healthcare provider.
• If you have any concerns or questions about diet while taking trofinetide, consult your healthcare provider or a registered dietitian for personalized guidance.

What should I do if I forget a dose?

• If you forget to take a dose of trofinetide, take it as soon as you remember unless it is almost time for your next scheduled dose. In that case, skip the missed dose and continue with your regular dosing schedule.
• Do not double up on doses to make up for a missed one, as this can increase the risk of side effects.
• If you are unsure about what to do or if you have missed multiple doses, contact your healthcare provider or pharmacist for advice.

What side effects can this medication cause?

As like other medication,  trofinetide can potentially cause side effects. Common side effects reported in clinical trials of trofinetide include:

• Gastrointestinal issues: Nausea, vomiting, diarrhea, and abdominal pain are among the most commonly reported gastrointestinal side effects.
• Fatigue: Some individuals may experience fatigue or tiredness as a side effect of trofinetide.
• Headache: Headaches are another commonly reported side effect.
• Dizziness: Some people may experience dizziness while taking trofinetide.
• Upper respiratory tract infections: In clinical trials, upper respiratory tract infections were reported as a common side effect.
• Decreased appetite: Trofinetide may cause a decrease in appetite in some individuals.
• Injection site reactions: For individuals receiving trofinetide through injections, reactions at the injection site such as redness, swelling, or pain may occur.

It’s important to note that not everyone will experience these side effects, and some individuals may experience side effects not listed here. Additionally, the severity of side effects can vary from person to person. It’s crucial for individuals taking trofinetide to discuss any concerns or side effects with their healthcare provider.

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

Storage and disposal of Trofinetide is important considerations to ensure safety and efficacy:

Storage:

• Follow Instructions: Store Trofinetide according to the instructions provided by the manufacturer or healthcare provider. This typically involves storing it at room temperature away from moisture, heat, and direct sunlight.
• Keep Out of Reach of Children: Store Trofinetide in a secure location out of reach of children and pets, as accidental ingestion can be harmful.
• Avoid Extreme Conditions: Avoid storing Trofinetide in locations with extreme temperatures, such as in the freezer or in a car during hot weather, as this can affect its stability and effectiveness.
• Proper Packaging: Keep Trofinetide in its original packaging or container to ensure proper identification and to prevent accidental misuse.

Disposal:

• Follow Local Regulations: Dispose of expired or unused Trofinetide in accordance with local regulations and guidelines. Some pharmacies or healthcare facilities may offer medication disposal programs to safely dispose of unused medications.
• Do Not Flush: Do not flush Trofinetide down the toilet or pour it down the drain unless instructed to do so by the manufacturer or healthcare provider.
• Consult Healthcare Provider: If you are unsure about how to dispose of Trofinetide safely, consult your healthcare provider or pharmacist for guidance.

In case of emergency/overdose

• Seek Medical Assistance: In case of an overdose or emergency, contact your local emergency services or poison control center immediately. Provide as much information as possible about the medication ingested, including the name, strength, and dosage.
• Symptom Management: If someone has overdosed on Trofinetide, monitor their symptoms closely and provide supportive care as needed while waiting for medical assistance to arrive.

What other information should I know?

• Follow Prescribing Instructions: Take Trofinetide exactly as prescribed by your healthcare provider. Do not adjust the dosage or frequency without consulting them first.
• Regular Monitoring: Your healthcare provider may need to monitor your response to Trofinetide regularly, including assessing for any side effects or changes in symptoms.
• Patient Education: Make sure to understand and follow any additional instructions provided by your healthcare provider regarding Trofinetide, including dietary restrictions, potential interactions with other medications, and signs of adverse reactions.
• Communication: Keep open communication with your healthcare provider and pharmacist regarding your experience with Trofinetide, including any concerns or questions you may have.

Always follow the specific instructions provided by your healthcare provider or pharmacist for the safe storage, disposal, and management of Trofinetide. If you have any questions or concerns, do not hesitate to seek guidance from a medical professional.