Trofinetide

Trofinetide is used to treat Rett syndrome (a genetic condition that causes severe developmental and nervous system problems). 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.

Trofinetide comes as a solution (liquid) or a powder to be mixed with liquid to take by mouth. Take with or without food twice a day. Take trofinetide at around the same times every day in the morning and the evening.

Take trofinetide exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Use an oral syringe or dosing cup (measuring device) to accurately measure and take your dose of trofinetide solution. Do not use a household spoon or cup. Ask your pharmacist for an oral syringe or dosing cup if one is not included with your medication.

To use the powder to be mixed with liquid:

1. measure the prescribed amount of liquid (cold to room temperature water or a water-based beverage like juice, tea, lemonaid, limeaid or liquid hydration solution) to make the dose and place in a cup
2. open and add all of the powder from the prescribed number of trofinetide packets to the measured liquid
3. stir until the powder is completely dissolved
4. take or give all of the prepared solution immediately. Do not store for later.
5. discard any prepared solution that is not taken right away

If you vomit after taking trofinetide, do not take another dose. Continue your regular dosing schedule the next day.

If you have a nasogastric (NG) tube, your doctor or pharmacist will explain how to give trofinetide solution through a NG tube.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before taking trofinetide,

• tell your doctor or pharmacist if you are allergic to this medication, any part of this medication, or any other medications, foods or substances. Tell your doctor or pharmacist about the allergy and what symptoms you had.
• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• the following nonprescription products may interact with trofinetide: laxatives. Be sure to let your doctor and pharmacist know that you are taking these medications before you start taking trofinetide. Your doctor will tell you to stop taking laxatives before you start taking trofinetide and not to take any laxative medications during your treatment.
• tell your doctor if you have or have ever had kidney problems.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking trofinetide, call your doctor.
• you should know that trofinetide can cause diarrhea, which can be severe. Your doctor may tell you to drink plenty of fluids or take other medications to control the diarrhea. Call your doctor immediately if you have severe diarrhea (diarrhea that does not stop) or diarrhea that does not get better after taking an anti-diarrhea medication. Call your doctor immediately if you experience any of the following symptoms: dry mouth, dark urine, decreased sweating, dry skin, and other signs of dehydration.
• you should know that trofinetide can cause vomiting which can lead to aspiration pneumonia. Call you doctor if vomiting is severe or persistent. Your doctor may tell you to hold, stop or decrease your dose of trofinetide.
• you should know that trofinetide can cause weight loss. Your doctor will monitor your weight closely during treatment with trofinetide. If you lose too much weight, your doctor may tell you to hold, stop or decrease your dose of trofinetide.

Unless your doctor tells you otherwise, continue your normal diet.

Skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.

Trofinetide may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• vomiting
• decreased appetite
• fever
• runny nose or sore throat
• unusual tiredness or weakness
• anxiety

Some side effects can be serious. If you experience any of these symptoms or those listed in the SPECIAL PRECAUTIONS section, call your doctor immediately or get emergency medical treatment:

• coughing, choking, shortness of breath, chest pain, or fever after vomiting
• seizures

Trofinetide may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store the bottle of trofinetide solution upright in a refrigerator, and dispose of any unused medication after 14 days. Do not freeze. Store trofinetide powder at room temperature and away from excess heat and moisture (not in the bathroom).

Keep all medication out of sight and reach of children as many containers are not child-resistant. Always lock safety caps. Place the medication in a safe location – one that is up and away and out of their sight and reach. https://www.upandaway.org

Dispose of unneeded medications in a way so that pets, children, and other people cannot take them. Do not flush this medication down the toilet. Use a medicine take-back program. Talk to your pharmacist about take-back programs in your community. Visit the FDA's Safe Disposal of Medicines website https://goo.gl/c4Rm4p for more information.

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Keep all appointments with your doctor.

Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.

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