Pronunciation:

troe-FIN-e-tide

Brand Names:

• Daybue

Dosage Forms:

• Solution

Classifications:

Therapeutic—

Central Nervous System Agent

Uses of This Medicine:

Trofinetide is used to treat Rett syndrome. Rett syndrome is a rare genetic disorder that affects the way the brain develops.

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of trofinetide in children younger than 2 years of age. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of trofinetide in the elderly. However, elderly patients are more likely to have age-related kidney problems, which may require caution in patients receiving this medicine.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Acalabrutinib
• Alfentanil
• Aprepitant
• Atorvastatin
• Avanafil
• Avapritinib
• Bosentan
• Bosutinib
• Budesonide
• Buspirone
• Cobimetinib
• Conivaptan
• Cyclosporine
• Darifenacin
• Darunavir
• Dasatinib
• Dihydroergotamine
• Dofetilide
• Dronedarone
• Elagolix
• Eletriptan
• Entrectinib
• Eplerenone
• Ergotamine
• Everolimus
• Felodipine
• Fentanyl
• Finerenone
• Flibanserin
• Fluticasone
• Fosaprepitant
• Glyburide
• Grazoprevir
• Ibrexafungerp
• Ibrutinib
• Indinavir
• Ivabradine
• Ivacaftor
• Letermovir
• Lomitapide
• Lovastatin
• Lurasidone
• Maraviroc
• Methotrexate
• Midazolam
• Naloxegol
• Oxycodone
• Pimavanserin
• Pimozide
• Pitavastatin
• Pravastatin
• Quetiapine
• Quinidine
• Repaglinide
• Revefenacin
• Rosuvastatin
• Sildenafil
• Simvastatin
• Sirolimus
• Sufentanil
• Tacrolimus
• Temsirolimus
• Ticagrelor
• Tipranavir
• Tolvaptan
• Triazolam
• Ubrogepant
• Vardenafil
• Venetoclax
• Voclosporin

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Kidney disease, moderate or severe—Use is not recommended.

Proper Use of This Medicine:

Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.

This medicine comes with a patient information leaflet. Read and follow the instructions carefully. Ask your doctor if you have any questions.

Measure the oral liquid medicine with a marked oral syringe or medicine cup. Do not use a household measuring cup.

You may also give this medicine using a gastrostomy tube.

You may take this medicine with or without food.

Do not take laxatives before starting treatment with this medicine.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage form (solution):
  • For Rett syndrome:
    • Adults and children 2 years of age and older—Dose is based on body weight and must be determined by your doctor.
      • Weighing 50 kilograms (kg) or more—12,000 milligrams (mg) or 60 milliliters (mL) 2 times a day. Your doctor may adjust your dose as needed.
      • Weighing 35 kg to less than 50 kg—10,000 mg or 50 mL 2 times a day. Your doctor may adjust your dose as needed.
      • Weighing 20 kg to less than 35 kg—8,000 mg or 40 mL 2 times a day. Your doctor may adjust your dose as needed.
      • Weighing 12 kg to less than 20 kg—6,000 mg or 30 mL 2 times a day. Your doctor may adjust your dose as needed.
      • Weighing 9 kg to less than 12 kg—5,000 mg or 25 mL 2 times a day. Your doctor may adjust your dose as needed.
    • Children younger than 2 years of age—Use and dose must be determined by your doctor.

Missed dose—

If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you vomit after taking your medicine, do not take an extra dose.

Storage—

Keep the bottle closed when you are not using it. Keep it in the refrigerator. Do not freeze.

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Throw away any unused medicine 14 days after opening the bottle.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure this medicine is working properly.

This medicine may cause severe diarrhea. Check with your doctor right away if you have fever, pain, stomach cramps, tenderness, or watery or bloody diarrhea. Your doctor will give you medicines to help prevent diarrhea during treatment with this medicine. Drink extra fluids to help prevent dehydration.

This medicine may cause weight loss. Talk to your doctor if you notice you are losing weight during treatment with this medicine.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Anxiety

decreased appetite

diarrhea

seizures

weight loss

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Fever

headache

muscle aches

sore throat

stuffy or runny nose

unusual tiredness or weakness

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.