Daybue
Daybue (trofinetide) is the first and currently only approved medication to treat Rett Syndrome in adults and children 2 years and older. It comes as a liquid solution which is helpful for people with Rett syndrome who have difficulty swallowing pills or use feeding tubes. The most common side effects of Daybue (trofinetide) include diarrhea and weight loss.
What is Daybue (trofinetide)?
What is Daybue (trofinetide) used for?
- Rett Syndrome in adults and children 2 years and older
How Daybue (trofinetide) works
Daybue (trofinetide) is a glycine-proline-glutamate analog, meaning it’s similar to a natural substance in our brain called glycine-proline-glutamate (GPE). How Daybue (trofinetide) helps with Rett Syndrome isn’t exactly clear, but it's thought that it works like GPE. This medication helps reduce inflammation, protects brain cells, and improves signals in the brain. As a result, it relieves symptoms of Rett Syndrome, like breathing difficulties, speech problems, and trouble making eye contact.
Drug facts
Side effects of Daybue (trofinetide)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Diarrhea (82%)
- Vomiting (29%)
- Weight loss (12%)
Less Common Side Effects
- Fever
- Seizure
- Anxiety
- Decreased appetite
- Fatigue
- Common cold
The following Daybue (trofinetide) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Dehydration—increased thirst, dry mouth, feeling faint or lightheaded, headache, dark yellow or brown urine
- Diarrhea
- Weight loss
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Loss of appetite
- Vomiting
Pros and cons of Daybue (trofinetide)
Pros
- Comes in a strawberry flavor, which is child-friendly
- Available in a liquid form for people who have difficulty swallowing pills or use feeding tubes
- Can take with or without food
- Can be used in children as young as 2 years old
Cons
- Not safe for people with moderate to severe kidney problems
- Commonly causes diarrhea and vomiting
- Must take the medication two times a day
- Might cause weight loss, which can worsen poor nutrition status in people with Rett syndrome
Pharmacist tips for Daybue (trofinetide)
- To get the right amount of Daybue (trofinetide) from the bottle, you must use a special syringe, cup, or dropper from the pharmacy. Ask your pharmacist for help so they can make sure you have the right tool to measure your medication correctly.
- Daybue (trofinetide) commonly causes diarrhea, and sometimes it can lead to dehydration. Signs of dehydration include feeling very thirsty, a dry mouth, and feeling tired. If this happens, let your child’s provider know. They can recommend drinking more fluids or taking anti-diarrhea medications. If the diarrhea doesn’t go away, the provider might lower the dose or even have your child stop Daybue (trofinetide).
- Taking Daybue (trofinetide) might make your child lose weight. It’s best to keep track of their weight after starting this medication. If you notice that they’re losing weight while taking Daybue (trofinetide), it's important to let your child’s provider know so they can help.
- Daybue (trofinetide) might interact and increase the level of other medications like carbamazepine (Tegretol), midazolam, and valsartan (Diovan). This can cause more unwanted side effects from these medications. Before your child starts any new medication or supplements, you should speak with their provider because they might change the dose or have your child avoid taking them together.
- Keep unopened and opened Daybue (trofinetide) bottles in the refrigerator at all times. After 14 days of opening the Daybue (trofinetide) bottle, throw away any leftover medication, even if there is leftover liquid. This is because the medication might not work as well if used after that time.
- If your child throws up after taking Daybue (trofinetide), don't give another dose to make up for it. Instead, just wait until it's time for their next dose and take it as usual.
- If you forget to give a dose of Daybue (trofinetide) to your child, just skip that dose and have them take the next dose at the usual time. Never take two doses to make up for the missed one since this can lead to more side effects from this medication.
Common questions about Daybue (trofinetide)
Rett Syndrome is a rare genetic condition that affects a child’s growth and development. It isn’t passed down from parents to their children, but it happens because of a random change (mutation) in a specific gene. This condition is mostly seen in girls. At first, a child’s growth seems to be on track with common developmental milestones. But later on, they can begin to lose their ability to talk or use their hands. Symptoms of Rett Syndrome vary from person to person but they might include trouble breathing, walking, eating, and overall delayed growth. While there is no cure for Rett Syndrome, Daybue (trofinetide) is the first and currently only FDA-approved medication to treat this condition.
No, you probably won't find Daybue (trofinetide) at your regular pharmacy. Instead, it will be sent to you from a specialty pharmacy that handles medications for rare conditions. You can find out more information about how to get this medication through Acadia Connect.
In a 12-week study, Daybue (trofinetide) improved eye contact, breathing, sleeping, and mood issues in some people with Rett Syndrome in as early as 6 weeks after starting the treatment. But the response to this medication can be different for each person since everyone reacts to medications in their own way. So it's important that your child takes Daybue (trofinetide) as prescribed at the same time every day in order to get the most relief from the medication.
Your child will take Daybue (trofinetide) by mouth or through feeding tubes twice daily, in the morning and evening. It can be taken with or without food. If your child has a gastrojejunal (GJ) tube, the gastric port (G-Port) must be used. However, you need to use a specifically marked syringe, cup, or dropper from the pharmacy to get the right amount of medication from the bottle. Don’t use household spoons or cups since they aren’t accurate.
People with moderate-to-severe kidney problems should avoid taking Daybue (trofinetide). This medication is mostly removed from the body by the kidneys. So if the kidneys aren't working well, the medication can stay in the body for longer and cause more side effects. Talk to the provider if you have concerns about kidney problems and Daybue (trofinetide).
There isn’t enough information in humans to support whether Daybue (trofinetide) is safe to use in pregnancy. In animal studies, Daybue (trofinetide) didn’t cause harm to the baby. But it’s important to note that even when the animals received the highest dose, the amount of this medication in their bodies was still lower than that found in humans taking the maximum dose. Talk with your provider if you’re pregnant or plan to get pregnant. They can make sure that Daybue (trofinetide) is safe for you and your baby.
Around 50% to 80% of people with Rett Syndrome have seizures, so it’s common for them to take anti-seizure medications. Daybue (trofinetide) might interact with other medications, including certain anti-seizure medications like carbamazepine (Tegretol) and midazolam. This can cause or worsen side effects from these seizure medications, such as dizziness and drowsiness. It’s important that before your child starts any new medication or supplements, you should talk with their provider. They'll help your child manage interactions with other medications.
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Risks and warnings for Daybue (trofinetide)
Daybue (trofinetide) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Diarrhea
- Risk factors: Taking laxative medications
Diarrhea is a very common side effect of Daybue (trofinetide). In the studies, around 85% of people who took Daybue (trofinetide) had mild to moderate diarrhea. Before starting this medication, make sure your child stops taking laxative medications that help with constipation.
If your child gets diarrhea while taking Daybue (trofinetide), tell their provider right away. One risk of having diarrhea is that your child can lose too much body fluids and become dehydrated. Symptoms of dehydration include feeling thirsty, tired, or dizzy. The provider might recommend that your child drinks more fluids to stay hydrated or takes certain anti-diarrhea medications. If the diarrhea doesn’t improve, your child’s provider might consider lowering the dose or stopping Daybue (trofinetide) altogether.
Weight loss
Taking Daybue (trofinetide) might cause your child to lose weight. In some cases, people can lose up to 7% or more of their body weight from before they started treatment. It’s important to keep an eye on your child’s weight by checking it every few days while taking this medication. If you notice any weight loss, let your child’s provider know. If your child loses a lot of weight, their provider might lower the dose or stop Daybue (trofinetide) completely.
Daybue (trofinetide) dosage
Typical dosage for Daybue (trofinetide)
Daybue (trofinetide) comes as an oral solution containing 200 mg per mL. Your child will take this medication by mouth or through a feeding tube two times a day, in the morning and evening, with or without food.
The starting dose will depend on your child’s weight:
- 9 kg to less than 12 kg: The typical dose is 5 grams (25 mL) twice daily.
- 12 kg to less than 20 kg: The typical dose is 6 grams (30 mL) twice daily.
- 20 kg to less than 35 kg: The typical dose is 8 grams (40 mL) twice daily.
- 35 kg to less than 50 kg: The typical dose is 10 grams (50 mL) twice daily.
- 50 kg or more: The typical dose is 12 grams (60 mL) twice daily.
The dose might differ if your child has side effects that don't go away, like diarrhea or weight loss.
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