Trofinetide
Trofinetide is a prescription oral medication used to treat Rett syndrome in adults and children age 2 and older.
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
📖 Trofinetide: Patient Information Guide
A plain-language walkthrough of Trofinetide — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Trofinetide is used to treat Rett syndrome, a rare neurological condition. Both branded forms carry this indication.
- Daybue (oral solution) is approved for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
- Daybue Stix (oral solution powder) is approved for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
⚙️How it works▾
The exact way trofinetide helps people with Rett syndrome is not yet known. Researchers have not fully identified the mechanism behind its therapeutic effects, though clinical studies have shown it is beneficial for patients with this condition.
💊How it's taken▾
Both Daybue and Daybue Stix are taken twice daily — once in the morning and once in the evening — and can be taken with or without food. The dose is based on body weight, so each patient's dose is individualized by their prescriber. Both products can be given by mouth or through a gastrostomy (G) tube.
- If you miss a dose, skip it and take your next dose at the regular time — do not double up.
- If vomiting occurs shortly after a dose, do not give an extra dose — continue with the next scheduled dose.
- Patients with moderate kidney impairment may need a lower dose; those with severe kidney impairment should generally not use these products.
🤒Side effects — in detail▾
The most common side effects reported during clinical trials include:
- Diarrhea — occurs in a very large proportion of patients
- Vomiting
- Fever
- Seizure
- Anxiety
- Decreased appetite
- Fatigue
- Nasopharyngitis (runny nose, cold-like symptoms)
Diarrhea and vomiting are the most important side effects to watch and manage promptly. Severe diarrhea with dehydration, or vomiting that leads to aspiration (inhaling fluid into the lungs), requires immediate medical attention and may mean stopping or adjusting the medication.
⚠️Warnings & precautions▾
- Diarrhea: About 85% of patients experience diarrhea on this medication. Stop laxatives before starting treatment. If diarrhea occurs, contact your doctor — antidiarrheal treatment and extra fluids may be needed. Severe or dehydrating diarrhea may require pausing or stopping the drug.
- Weight loss: Meaningful weight loss can occur. Weight should be monitored regularly, and significant loss may require a dose change or stopping treatment.
- Vomiting and aspiration risk: Vomiting occurs in roughly 1 in 4 patients. Because people with Rett syndrome are already at higher risk for aspiration (breathing in food or liquid), vomiting has led to aspiration pneumonia in some patients. Report severe or repeated vomiting to your doctor right away.
🔀What it interacts with▾
Trofinetide can affect the blood levels of other medications you take. The most clinically important interaction involves drugs that are broken down by the CYP3A enzyme or transported by P-gp in the body.
- CYP3A and/or P-gp substrates: Trofinetide can raise blood levels of these drugs, which may increase the chance of serious side effects from those medications. Your doctor should monitor you closely if you take any of these.
- Laxatives: Stop all laxatives before starting trofinetide, as directed by your doctor, to help reduce the risk of severe diarrhea.
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Kidney disease: If you have moderate kidney impairment (reduced kidney filtering ability), your dose will need to be lowered. If you have severe kidney impairment, this drug is generally not recommended.
- Older adults: Older patients are more likely to have reduced kidney function; kidney function monitoring may be useful.
- Pregnancy: There are no adequate human safety data during pregnancy. Animal studies did not show harm, but those exposures were lower than typical human doses. Discuss the risks and benefits with your doctor.
- Breastfeeding: It is not known whether trofinetide passes into breast milk or affects a breastfed baby. Talk to your doctor about whether to continue breastfeeding while on this medication.
- Children under 2: Safety and effectiveness have not been established in patients younger than 2 years of age.
- Liver disease: Trofinetide is not significantly processed by the liver, so liver problems are not expected to have a meaningful impact on how this drug works in the body.
📡Pharmacodynamics — effects in the body▾
At the highest recommended dose, trofinetide does not meaningfully prolong the QT interval (a measure of heart electrical activity) in healthy adults, which means it is not expected to cause a type of irregular heart rhythm associated with QT prolongation.
🔬Pharmacokinetics — how your body processes it▾
After you take trofinetide by mouth, peak drug levels in the blood are reached in about 2 to 3 hours, and at least 84% of the dose is absorbed. It is not significantly broken down by the liver — instead, about 80% of the drug is cleared from the body unchanged through the urine. The effective half-life (the time it takes for half the drug to leave your system) is about 1.5 hours, and the drug does not significantly build up in the body with repeated doses. Taking it with a high-fat meal does not change the total amount absorbed, though peak levels may be slightly lower.
📦How to store it▾
Store DAYBUE in an upright position refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. Store DAYBUE STIX packets at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (59°F and 86°F) .
📄 Written from Trofinetide’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
Supplements & herbs that interact with Trofinetide
30 supplements and herbal products have documented interactions with Trofinetide in the licensed clinical database we use. Most are manageable — but your pharmacist should know about everything you take, including vitamins and herbals.
- Major · 1
- Moderate · 19
- Minor · 10
Educational information from a licensed clinical database (Natural Medicines) — see our data sources & update cadence. Not medical advice: an interaction being documented does not mean it will happen to you; do not start or stop medications or supplements without professional guidance.
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Trofinetide — the kind of thing our pharmacy team would talk through with you at the counter.
What is trofinetide actually used for? ▾
How do I take it, and does it matter if I take it with food? ▾
What about Daybue Stix — how is that different from regular Daybue? ▾
What side effects should I really watch for? ▾
Does trofinetide interact with other medications? ▾
Is it safe to use if my child has kidney problems, or if I'm pregnant? ▾
Is Trofinetide available over the counter? ▾
When was Trofinetide first available? ▾
Who makes Trofinetide? ▾
💬 Answers drafted from Trofinetide’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Trofinetide comes
Trofinetide is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Oral solution
How this form is used
- Daybue oral solution is indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older
- Daybue Stix for oral solution is indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older
- Daybue oral solution and Daybue Stix are both taken by mouth twice daily (morning and evening), with or without food, and can also be given via a gastrostomy (G) tube
- Daybue oral solution should be measured with a calibrated device (such as an oral syringe or dosing cup) obtained from the pharmacy — do not use a household spoon
- Daybue Stix powder must be dissolved in cold to room-temperature water or a water-based beverage (juice, tea, lemonade, limeade, or liquid hydration) right before use and administered immediately — do not store the prepared solution
- Daybue oral solution should be discarded 14 days after first opening the bottle; unused Daybue Stix prepared solution that is not immediately given should be discarded
📊 Trofinetide across the U.S. market
How Trofinetide appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
Also listed with the FDA, not a patient dose form: Powder (8) — bulk drug substance sold to compounders/manufacturers and multi-item kits. These aren’t counted in the dose-form total above.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
📈 How widely Trofinetide is used
A general measure of how commonly Trofinetide is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Trofinetide is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Trofinetide prescribed? Of the 1,596 medications we measure, Trofinetide ranks #1,055 by Medicaid prescriptions — more than 34% of them.
Utilization by Trofinetide product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
trofinetide 200 MG/ML Oral Solution
Summed across the 1 of 1 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 2632857
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Trofinetide, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 1 of 1 listed Trofinetide NDCs with Medicaid activity.
🔍 Compare Trofinetide products
A representative set of FDA-listed product records for Trofinetide — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Oral solution | ||||
| 200 mg/mL | Oral | Acadia Pharmaceuticals Inc. × 4 listings | NDA | View NDC → |
| 5000 mg | Oral | Acadia Pharmaceuticals Inc. × 3 listings | NDA | View NDC → |
| 6000 mg | Oral | Acadia Pharmaceuticals Inc. × 3 listings | NDA | View NDC → |
| 8000 mg | Oral | Acadia Pharmaceuticals Inc. × 3 listings | NDA | View NDC → |
| 💊Bulk drug substance | ||||
| 50 kg/50 kg | — | Aurobindo Pharma Limited | BULK INGREDIENT | View NDC → |
| 50 kg/50 kg | — | Biophore India Pharmaceuticals Pvt. Ltd | BULK INGREDIENT | View NDC → |
| 1 kg/kg | — | Corden Pharma Bergamo SpA | BULK INGREDIENT | View NDC → |
| 1 kg/kg | — | F.i.s. Fabbrica Italiana Sintetici S.p.a. × 2 listings | BULK INGREDIENT | View NDC → |
| 1 kg/kg | — | Flamma S.p.a. × 3 listings | BULK INGREDIENT | View NDC → |
Showing 21 of 21 products — FDA National Drug Code Directory. See every product, package size and price: browse all 21 Trofinetide NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Trofinetide — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Trofinetide in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Trofinetide went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2632852 2 dose forms · 4 strengths
-
Dose form (SCDF) Oral Solution RxCUI 2632856In current FDA listingsClinical drug / strength (SCD) 200 MG/MLRxCUI 2632857 -
Dose form (SCDF) Powder for Oral Solution RxCUI 2729446In current FDA listings
Look up RxCUI 2632852 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Trofinetide is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
Trofinetide belongs to the Other nervous system drugs class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
For canonical ingredient pages, bounded product-attributed indication and administration excerpts may be added to the AI evidence, and the generator is instructed to preserve available product, brand, route, and form qualifiers instead of treating one label as universal. Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Acadia Pharmaceuticals Inc. is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.