Trofinetide is for use in adults and children at least 2 years old to treat Rett syndrome.

Trofinetide may also be used for purposes not listed in this medication guide.

Use only as directed. Tell your doctor if you use other medicines or have other medical conditions or allergies.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

If you take laxatives, stop taking them before starting trofinetide.

Trofinetide is usually taken twice per day, in the morning and evening, with or without food.

Trofinetide can also be given through a gastrostomy feeding tube. Follow your doctor's instructions.

Doses are based on weight. Your dose may change if you gain or lose weight.

Measure liquid medicine with the supplied measuring device (not a kitchen spoon).

If you vomit shortly after taking trofinetide, do not take another dose. Take your next dose as scheduled.

Your kidney function may need to be checked often.

Your treatment with trofinetide may be interrupted, changed, or discontinued if you have certain side effects.

Store tightly closed in an upright position in the refrigerator, do not freeze.

Throw the medicine away after 14 days of first opening the bottle, even if there is still medicine left inside.

Usual Adult Dose for Rett Syndrome:

Oral:
18 years or older:
-Weight 9 to less than 12 kg: 5,000 mg (25 mL) orally twice a day
-Weight 12 to less than 20 kg: 6,000 mg (30 mL) orally twice a day
-Weight 20 to less than 35 kg: 8,000 mg (40 mL) orally twice a day
-Weight 35 to less than 50 kg: 10,000 mg (50 mL) orally twice a day
-50 kg or more: 12,000 mg (60 mL) orally twice a day

Comments:
-This drug can be taken with or without food twice daily, in the morning and evening.
-If a dose is missed, an additional dose should not be taken to make up for the missed dose. Instead, wait and continue with the next scheduled dose.
-In the event of vomiting, do not take another dose to make up for that dose. Wait and take the following dose as scheduled.

Use: For the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older

Usual Pediatric Dose for Rett Syndrome:

Oral:
2 to less than 18 years:
-Weight 9 to less than 12 kg: 5,000 mg (25 mL) orally twice a day
-Weight 12 to less than 20 kg: 6,000 mg (30 mL) orally twice a day
-Weight 20 to less than 35 kg: 8,000 mg (40 mL) orally twice a day
-Weight 35 to less than 50 kg: 10,000 mg (50 mL) orally twice a day
-50 kg or more: 12,000 mg (60 mL) orally twice a day

Comments:
-This drug can be taken with or without food twice daily, in the morning and evening.
-If a dose is missed, an additional dose should not be taken to make up the missed dose. Instead, wait and continue with the next scheduled dose.
-In the event of vomiting, do not take another dose to make up for that dose. Wait and take the following dose as scheduled.

Use: For the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older

Tell your doctor if you have or have ever had:

• if you use a laxative; or

• kidney disease.

It is not known if trofinetide will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Ask a doctor if it is safe to breastfeed while using trofinetide.

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

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. http://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 (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

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

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Trofinetide may cause serious side effects. Call your doctor at once if you have:

• severe diarrhea;

• weight loss; or

• dehydration--dizziness, confusion, feeling very thirsty, less urination.

Common side effects may include diarrhea or vomiting.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Other drugs may affect trofinetide, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

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.