Trofinetide

Trofinetide is used to treat Rett syndrome (a genetic 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.

Trofinetide comes as an solution (liquid) to take by mouth. It is usually taken twice a day, in the morning and in the evening, with or without food. Take trofinetide at around the same times every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. 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 cup if one is not included with your medication.

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 and pharmacist if you are allergic to trofinetide, any other medications, or any of the ingredients in trofinetide solution. Ask your pharmacist for a list of the ingredients.
• tell your doctor if you are taking laxatives. Your doctor will tell you to stop taking laxatives before you start taking trofinetide and not to take any laxative medications while you are taking trofinetide.
• 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.
• 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.

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, sore throat
• tiredness
• anxiety

Some side effects can be serious. If you experience any of these symptoms, stop taking trofinetide and call your doctor immediately or get emergency medical treatment:

• diarrhea
• dry mouth, dark urine, decreased sweating, dry skin, and other signs of dehydration
• weight loss

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 upright in a refrigerator, and dispose of any unused medication after 14 days. Do not freeze.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. https://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (https://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

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.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

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