Gilteritinib
Pronunciation: pronunciation varies by local usage.
- Generic name
- gilteritinib
- Brand names
- XOSPATA
- Drug class
- FLT3 kinase inhibitor
- Form
- Film-coated tablets (40 mg)
- Route
- Oral
- Legal status
- Prescription-only medicine
Availability and supply rules vary by country
Trade names by country: United States
Australia: · Full directory
What gilteritinib is and how it works
Gilteritinib is an oral kinase inhibitor that targets FLT3, used in adults with relapsed or refractory acute myeloid leukemia (AML) that has a FLT3 mutation as detected by an FDA-approved test. (1)
Treatment is initiated and monitored by a specialist. It is taken once daily for at least 6 months to allow time for response, with regular blood tests and ECGs required because of risks including differentiation syndrome, QT prolongation, and pancreatitis.
Mechanism of action: how gilteritinib works
Gilteritinib is a small molecule FLT3 kinase inhibitor that blocks FLT3 receptor signaling, inhibiting proliferation of leukemic cells expressing FLT3-ITD or FLT3-TKD mutations. (1)
What gilteritinib is used for
Relapsed or refractory acute myeloid leukemia with a FLT3 mutation
Gilteritinib is indicated for adults with relapsed or refractory AML that has a FLT3 mutation as detected by an FDA-approved test. Treatment should be continued for a minimum of 6 months to allow clinical response. (1)
Before taking gilteritinib
Do not take / not appropriate for
- Do not take gilteritinib if you have had a severe allergic reaction to gilteritinib or any of its ingredients unless your specialist determines otherwise after careful assessment. (all products) (1)
Tell your clinician about
- All current medicines, especially blood thinners, other cancer treatments, or medicines that can affect heart rhythm (e.g., escitalopram, fluoxetine, sertraline).
- Any history of heart problems, seizures, liver or kidney conditions.
- If you are pregnant, planning to become pregnant, or breastfeeding.
- Any new or worsening symptoms such as fever, breathlessness, rapid weight gain, swelling, low blood pressure, or confusion.
- Whether you have a known FLT3 mutation confirmed by an FDA-approved test.
How to take gilteritinib
- Take gilteritinib exactly as prescribed by your specialist. The usual dose is 120 mg (three 40 mg tablets) taken orally once daily.
- Treatment is usually continued for at least 6 months to allow time for a response. Do not stop taking it or change the dose without your doctor's instruction.
- You will need regular blood tests and ECGs as directed by your doctor to monitor for side effects.
- Swallow the tablets whole with a glass of water, at the same time each day.
Procedures and treatment changes
Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (1)
Missed dose
No specific missed dose guidance is provided in the current US label. If a dose is missed, contact your doctor for instructions. Do not take an extra dose to make up for a missed one.
Overdose
No specific overdose information is provided in the current US label. If an overdose is suspected, contact your healthcare provider or poison control center immediately. Seek emergency care for any serious symptoms. (1)
Side effects
Gilteritinib can cause serious side effects, including differentiation syndrome (a boxed warning), QT prolongation, pancreatitis, and PRES. Common side effects include changes in liver and other blood tests, muscle or joint pain, tiredness, fever, mouth sores, swelling, rash, diarrhea, and shortness of breath.
Increase in liver enzymes (transaminase)
What to notice: May not cause symptoms; routine blood tests monitor liver function.
What to do: Your doctor will check blood tests regularly; report any signs of liver problems like yellowing of skin/eyes, dark urine, or right upper abdominal pain. (1)
Muscle or joint pain
What to notice: Aching, stiffness, or discomfort in muscles or joints.
What to do: Mention persistent or troublesome pain to your doctor; they can advise on management. (1)
Fatigue, fever, or mouth sores
What to notice: Unusual tiredness, elevated body temperature, or painful sores in the mouth.
What to do: Contact your doctor if these symptoms are severe or do not improve; blood counts may need to be checked. (1)
Swelling (edema) or rash
What to notice: Swelling of the hands, feet, or other body parts; skin changes including redness or itching.
What to do: Inform your doctor if swelling is sudden or severe, or if rash is extensive or accompanied by other symptoms. (1)
Diarrhea or nausea
What to notice: Frequent loose stools or feeling sick to the stomach.
What to do: Stay hydrated; tell your doctor if diarrhea is severe or persistent. (1)
Symptoms of pancreatitis
What to notice: Severe upper abdominal pain that may spread to the back, nausea, vomiting.
What to do: Contact your doctor immediately or seek emergency care. (1)
Symptoms of PRES
What to notice: Seizure, severe headache, confusion, vision changes.
What to do: Seek emergency medical attention. (1)
Heart rhythm changes (QT prolongation)
What to notice: Feeling faint, dizzy, irregular heartbeat, palpitations.
What to do: Contact your doctor promptly. Do not adjust your dose yourself; your doctor may arrange an ECG and check electrolytes. (1)
Signs of liver or kidney problems
What to notice: Yellowing of skin or eyes, dark urine, severe tiredness, pain in the upper right belly, decreased urine output.
What to do: Contact your doctor for assessment. (1)
Serious warnings
Differentiation syndrome (boxed warning) — emergency
Symptoms: Fever, cough, trouble breathing, low oxygen, fluid around lungs or heart, rapid weight gain, swelling, low blood pressure, kidney failure.
Action: Seek immediate medical attention. The treating team may need to interrupt gilteritinib and administer corticosteroids. Do not delay reporting these symptoms. (1)
QT prolongation — urgent
Symptoms: Feeling faint, dizzy, irregular heartbeat, palpitations.
Action: Contact your doctor immediately. Your doctor may monitor your ECG and electrolytes and may adjust the dose. (1)
Pancreatitis — urgent
Symptoms: Severe upper abdominal pain, nausea, vomiting.
Action: Seek medical attention promptly. Your doctor may evaluate and potentially interrupt treatment. (1)
PRES — emergency
Symptoms: Seizure, severe headache, confusion, vision changes.
Action: Seek emergency medical care. Gilteritinib may need to be permanently discontinued; this decision will be made by your specialist. (1)
Interactions
| Medicine or test | Effect | Action |
|---|---|---|
| Combined P-glycoprotein (P-gp) and strong CYP3A inducers (e.g., rifampin, carbamazepine, phenytoin) | Significantly reduces gilteritinib exposure, which may decrease effectiveness. | Avoid co-administration. Your doctor will consider alternative therapies. (1) |
| Strong CYP3A inhibitors (e.g., ketoconazole, clarithromycin) | Increases gilteritinib exposure, raising the risk of side effects. | Consider alternative medicines or increase monitoring frequency. Your doctor will evaluate the risks and benefits. (1) |
| Drugs that are substrates of P-gp, BCRP, or OCT1 (e.g., digoxin, methotrexate) | Gilteritinib may increase their levels, potentially leading to toxicity. | Your doctor may decrease the dose of the co-administered drug. (1) |
| Drugs targeting 5-HT2B or sigma receptors (e.g., escitalopram, fluoxetine, sertraline) | Possible additive effects or increased risk of side effects. | Avoid unless essential. Every medicine must be reviewed by your doctor. (1) |
Pregnancy, breastfeeding, kidney/liver function, age
Pregnancy and breastfeeding
Gilteritinib can cause fetal harm. Pregnancy testing is required within 7 days before starting. Effective contraception must be used by females during treatment and for 6 months after the last dose, and by males during treatment and for 4 months after. Breastfeeding is not recommended during treatment and for 2 months after the final dose. (1)
Children and adolescents
Safety and efficacy have not been established in pediatric patients. Gilteritinib is indicated for adults only. (1)
Patients with kidney or liver impairment
The current label does not provide specific dose adjustments for renal or hepatic impairment. Clinical monitoring and specialist judgment are necessary. (1)
Monitoring
Before starting
- Confirm the presence of a FLT3 mutation with an FDA-approved test.
- Perform blood counts, blood chemistries (including liver and renal function), and creatine kinase.
- Assess baseline ECG, and correct any electrolyte abnormalities (potassium, magnesium).
- Evaluate for pregnancy in women of childbearing potential.
- Review all concomitant medications for potential interactions.
During treatment
- Blood counts, blood chemistries, and creatine kinase: weekly during the first month, every other week in month 2, then monthly thereafter.
- ECG: before starting, on days 8 and 15 of cycle 1, and before the start of the subsequent two cycles.
- Monitor closely for signs and symptoms of differentiation syndrome, especially during the first 3 months.
- Monitor for QTc prolongation and electrolyte disturbances; replete potassium and magnesium as needed.
- Assess for pancreatitis, PRES, and other serious adverse events throughout treatment.
Storage
Store at 20–25°C (68–77°F); excursions permitted to 15–30°C (59–86°F). Keep in the original container to protect from light and moisture.
Professional information
Clinician-facing context only. Use the current local prescribing information and guidelines for relapsed/refractory AML.
United States
Indication: Relapsed or refractory AML with FLT3 mutation
Dose summary: 120 mg orally once daily. Treat for a minimum of 6 months. Dose modifications by the specialist: discontinue for PRES; interrupt and resume at 80 mg for QTcF >500 ms, pancreatitis, or other Grade ≥3 toxicity.
Renal context: No specific dose adjustment provided in label. Use with caution and monitor closely. (1)
Hepatic guidance
No specific dose adjustment provided for hepatic impairment. Monitor liver function.
Overdose note
No specific overdose information is available. Symptomatic and supportive measures should be used as appropriate. (1)
Frequently asked questions
What is differentiation syndrome and why is it in a boxed warning?
Differentiation syndrome is a potentially fatal reaction that can occur with gilteritinib. Symptoms include fever, breathing trouble, rapid weight gain, swelling, and low blood pressure, and they can start 1–82 days after beginning treatment. The boxed warning is the strongest FDA warning. The treating team manages it with corticosteroids and may interrupt gilteritinib. Any symptoms must be reported immediately. (1)
Why do I need repeated ECGs and blood tests?
Gilteritinib can affect your heart rhythm (prolong the QT interval) and alter blood counts, liver enzymes, and creatine kinase. ECGs are done before treatment, on days 8 and 15 of the first cycle, and before the next two cycles. Blood tests are checked weekly in the first month, every other week in the second month, then monthly. This monitoring helps your doctor manage side effects promptly. (1)
How long will I take gilteritinib?
The label recommends treating for at least 6 months to allow a response. The median time to first response is about 2–3.6 months. Your specialist will decide the total duration based on how well the leukemia responds and how you tolerate the treatment. (1)
Can I take antidepressants with gilteritinib?
Medicines that target 5-HT2B or sigma receptors, such as escitalopram, fluoxetine, or sertraline, should be avoided unless essential. You must discuss all your current medicines, including antidepressants, with your treatment team so they can assess the risks. (1)
What is the mechanism of action of gilteritinib?
Gilteritinib is a small molecule FLT3 kinase inhibitor that blocks FLT3 receptor signaling, inhibiting proliferation of leukemic cells expressing FLT3-ITD or FLT3-TKD mutations. (1)
What is the generic name of Gilteritinib?
The generic name is gilteritinib. It is sold under brand names including XOSPATA. (1)
What class of drug is gilteritinib?
Gilteritinib is classed as: FLT3 kinase inhibitor. (1)
References
- Xospata- gilteritinib tablet, film coatedDailyMed (U.S. National Library of Medicine) · United States · dated 2022-01 · accessed 2026-08-08 · source 1
- Gilteritinib — patient drug information (MedlinePlus)U.S. National Library of Medicine (MedlinePlus) · US · accessed 2026-08-14 · source 2
- Xospata — FDA approval record (Drugs@FDA NDA211349)U.S. Food and Drug Administration · US · accessed 2026-08-14 · source 3
- gilteritinib — RxNorm normalized drug concept (RXCUI 2105806)U.S. National Library of Medicine (RxNorm) · US · accessed 2026-08-14 · source 4
Brand names by country
Single-ingredient gilteritinib names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.
Priority countries
United States
- Xospata40 mg · astellas pharma us, inc.