Xospata (gilteritinib)
An approved treatment for Acute Myeloid Leukemia.
The same compound appears under different names depending on the context. Here is how to identify Gilteritinib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Gilteritinib
- Brand name
- Xospata
- Development code
- ASP2215
- Drug class
- Kinase inhibitor (FLT3, type I)
- Manufacturer
- Astellas Pharma
- How it's taken
- Oral tablets once daily
A once-daily pill for adults whose FLT3-mutated AML has relapsed or never responded to treatment. In the ADMIRAL trial it roughly doubled the chance of remission and lengthened survival compared with salvage chemotherapy. Approved November 28, 2018.
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Where Gilteritinib fits
The standard targeted option at relapse for FLT3-mutated AML, used alone, and often as a bridge to transplant. Trials are testing it combined with venetoclax, azacitidine and menin inhibitors, and as maintenance after transplant.
How Gilteritinib works
A FLT3 mutation keeps a growth receptor on the leukemia cell permanently switched on. Gilteritinib blocks the FLT3 receptor directly, including the ITD form and the TKD point mutations at D835 and I836 that can make leukemia resistant to other FLT3 drugs, and it also blocks AXL, a related receptor linked to resistance[1]. Unlike Rydapt and Vanflyta, which are added to chemotherapy, Xospata is taken on its own.
Mechanism: Oral FLT3 inhibitor active against FLT3-ITD and FLT3-TKD (D835, I836) mutations, with AXL activity
Side effects and safety
- Posterior reversible encephalopathy syndrome. In 1%; seizures or altered mental state, confirmed by brain imaging; Xospata is stopped for good.
- QT prolongation. ECG before starting, on days 8 and 15 of cycle 1 and before cycles 2 and 3; interrupt and reduce if QTcF goes above 500 msec; keep potassium and magnesium normal.
- Pancreatitis. Interrupt and reduce the dose if pancreatitis develops.
- Embryo-fetal toxicity. Can harm an unborn baby; contraception during treatment and for 6 months after (women) or 4 months after (men).
- Blood counts and chemistry including creatine phosphokinase before starting and at least weekly for the first month, every other week for the second month, then monthly
- ECG on the schedule above
- Liver tests
- Watch for signs of differentiation syndrome for the first weeks
Xospata carries a boxed warning for differentiation syndrome, which occurred in 3% of 319 trial patients between 1 and 82 days after starting; symptoms include fever, breathlessness, fluid around the lungs or heart, rapid weight gain, low blood pressure and kidney problems, and it is treated with dexamethasone[1]. Other warnings are posterior reversible encephalopathy syndrome (1%, with seizures or confusion), QT prolongation (interrupt if QTcF exceeds 500 msec; correct potassium and magnesium), pancreatitis and harm to an unborn baby[1].
The most common side effects (20% or more) were raised liver enzymes (51%), muscle or joint pain (50%), fatigue (44%), fever (41%), mouth sores (41%), swelling (40%), rash (36%), diarrhea (35%), shortness of breath (35%), nausea (30%), cough, constipation, eye problems, headache, dizziness, low blood pressure, vomiting and kidney impairment; 7% stopped because of side effects and fatal adverse reactions occurred in 2%[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Gilteritinib
Three 40 mg tablets (120 mg) by mouth once a day, with or without food, at about the same time each day. Response can take time, so the label recommends at least 6 months of treatment if the disease is not progressing[1]. Tablets are not crushed; a missed dose is taken the same day if at least 12 hours remain before the next one. Treatment continues until the leukemia progresses or side effects become unacceptable, and people who reach remission may go on to a transplant[1].
Availability and cost
Only available as the brand-name product.
Help paying for Xospata
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
XOSPATA Patient Savings Program: eligible commercially insured patients pay as little as $0 per prescription, enrolled for 12 months, with a maximum of $7,000 per calendar year and no income requirement. Not for Medicaid, Medicare, Medigap, DoD, VA, TRICARE or state assistance programs; the benefit may drop to $25 per claim if your plan uses a maximizer or accumulator.
For: private insurance · source - Free medicine program
Astellas Patient Assistance Program provides Xospata at no cost to patients who are uninsured or whose insurance excludes it, have a US shipping address, are prescribed it for an FDA-approved use and meet financial criteria. The doctor's office submits the application.
For: no insurance, underinsured · source - Insurance and case manager help
Dedicated access specialists at 1-844-632-9272, Monday to Friday, 8 AM to 8 PM ET, for benefit verification, coverage questions and Medicare resources.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Copay enrollment is at XOSPATAcopayenroll.com.
Access and eligibility
Adults with relapsed or refractory AML and a FLT3 mutation detected by an FDA-approved test (the LeukoStrat CDx assay). It is not approved for newly diagnosed AML, where Rydapt and Vanflyta are the FLT3 options, nor as post-transplant maintenance.
Source: Astellas Pharma Support Solutions
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
How the targeted AML pills compare, by mutation
AML has 19 FDA-approved medicines, counting the 3 older chemotherapy drugs. This chart covers the 8 that target a specific gene change, because the result of your mutation test decides which of them could apply to you. Chemotherapy (7+3, Vyxeos), Venclexta, Mylotarg, Onureg, Daurismo and the APL drugs are described on the drug pages instead. Several of these drugs are only approved after relapse, and all of the FLT3, IDH and menin drugs can cause differentiation syndrome, so your leukemia doctor will weigh the mutation, your point in treatment and your heart and liver tests.
Acute Myeloid Leukemia has 8 FDA-approved treatments: Rydapt (midostaurin, approved 2017); Vanflyta (quizartinib, approved 2023); Xospata (gilteritinib, approved 2018); Tibsovo (ivosidenib, approved 2018); Rezlidhia (olutasidenib, approved 2022); Idhifa (enasidenib, approved 2017); Revuforj (revumenib, approved 2024); Komzifti (ziftomenib, approved 2025). Rydapt is typically used as newly diagnosed, with intensive chemotherapy.
| Drug | Mutation it targets | When it is approved | How it's taken | How often | Given with | FDA approved |
|---|---|---|---|---|---|---|
Rydapt midostaurin | FLT3 (ITD or TKD) | Newly diagnosed, with intensive chemotherapy | Oral capsule | Twice daily on days 8 to 21 of each chemotherapy cycle | 7+3 induction and high-dose cytarabine consolidation (hospital-based chemotherapy) | 2017 |
Vanflyta quizartinib | FLT3-ITD only | Newly diagnosed, with intensive chemotherapy, then maintenance | Oral tablet | Once daily, with chemotherapy and then up to 36 months of maintenance | 7+3 induction and cytarabine consolidation, then alone as maintenance (not after transplant) | 2023 |
Xospata gilteritinib You are here | FLT3 (ITD or TKD) | Relapsed or refractory | Oral tablets | Once daily | Taken alone | 2018 |
Tibsovo ivosidenib | IDH1 | Newly diagnosed (older or unfit) and relapsed or refractory | Oral tablets | Once daily | With azacitidine or alone (newly diagnosed, 75+ or unfit); alone at relapse | 2018 |
Rezlidhia olutasidenib | IDH1 | Relapsed or refractory | Oral capsule | Twice daily on an empty stomach | Taken alone | 2022 |
Idhifa enasidenib | IDH2 | Relapsed or refractory | Oral tablet | Once daily | Taken alone | 2017 |
Revuforj revumenib | KMT2A rearrangement (any acute leukemia) or NPM1 mutation (AML) | Relapsed or refractory (NPM1: after other options) | Oral tablets | Twice daily | Taken alone; adults and children 1 year and older | 2024 |
Komzifti ziftomenib | NPM1 mutation | Relapsed or refractory, after other options | Oral capsules | Once daily on an empty stomach | Taken alone; adults only | 2025 |
This chart is for education only and is not medical advice. Approval dates, indications and warnings come from each drug's FDA prescribing information and Drugs@FDA. None of these drugs has been compared head to head with another in the same column, and trial results are not comparable across different studies. A leukemia specialist who has your mutation report will know which, if any, applies to you and whether a clinical trial would fit better.
Clinical trial results
ADMIRAL (NCT02421939) randomized 371 adults with relapsed or refractory FLT3-mutated AML 2 to 1 to gilteritinib or the investigator's choice of salvage chemotherapy (high-intensity MEC or FLAG-IDA, or low-intensity cytarabine or azacitidine). Median overall survival was 9.3 months with gilteritinib against 5.6 months with chemotherapy (hazard ratio 0.64, 95% CI 0.49 to 0.83, p=0.0004).
Complete remission was 14.2% against 10.5%, and the complete remission or partial-count-recovery rate on gilteritinib was 22.6% (55/243) with a median duration of 7.4 months[1][2]. Responses were seen in people with FLT3-ITD or ITD plus TKD mutations; in the first interim analysis none of the 12 patients with a TKD mutation alone responded[1].
Development history
Explore Acute Myeloid Leukemia trials
Other Acute Myeloid Leukemia treatments
Common questions about Gilteritinib
▸What is Gilteritinib (Xospata)?
A once-daily pill for adults whose FLT3-mutated AML has relapsed or never responded to treatment. In the ADMIRAL trial it roughly doubled the chance of remission and lengthened survival compared with salvage chemotherapy. Approved November 28, 2018.
▸How does Gilteritinib work?
A FLT3 mutation keeps a growth receptor on the leukemia cell permanently switched on. Gilteritinib blocks the FLT3 receptor directly, including the ITD form and the TKD point mutations at D835 and I836 that can make leukemia resistant to other FLT3 drugs, and it also blocks AXL, a related receptor linked to resistance[1]. Unlike Rydapt and Vanflyta, which are added to chemotherapy, Xospata is taken on its own.
▸What are the side effects of Gilteritinib?
Xospata carries a boxed warning for differentiation syndrome, which occurred in 3% of 319 trial patients between 1 and 82 days after starting; symptoms include fever, breathlessness, fluid around the lungs or heart, rapid weight gain, low blood pressure and kidney problems, and it is treated with dexamethasone[1]. Other warnings are posterior reversible encephalopathy syndrome (1%, with seizures or confusion), QT prolongation (interrupt if QTcF exceeds 500 msec; correct potassium and magnesium), pancreatitis and harm to an unborn baby[1]. The most common side effects (20% or more) were raised liver enzymes (51%), muscle or joint pain (50%), fatigue (44%), fever (41%), mouth sores (41%), swelling (40%), rash (36%), diarrhea (35%), shortness of breath (35%), nausea (30%), cough, constipation, eye problems, headache, dizziness, low blood pressure, vomiting and kidney impairment; 7% stopped because of side effects and fatal adverse reactions occurred in 2%[1].
▸How is Gilteritinib taken?
Three 40 mg tablets (120 mg) by mouth once a day, with or without food, at about the same time each day. Response can take time, so the label recommends at least 6 months of treatment if the disease is not progressing[1]. Tablets are not crushed; a missed dose is taken the same day if at least 12 hours remain before the next one. Treatment continues until the leukemia progresses or side effects become unacceptable, and people who reach remission may go on to a transplant[1].
▸Is Gilteritinib FDA approved?
Yes, Gilteritinib (Xospata) is FDA approved (2018) for the treatment of Acute Myeloid Leukemia.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Astellas Pharma US via DailyMed · 2026-10-01. Xospata (gilteritinib) tablets prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5ff59aa-9c0d-49a8-9053-1f179b482383
- New England Journal of Medicine · 2019-10-31. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. https://pubmed.ncbi.nlm.nih.gov/31665578/
- FDA. Xospata approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=211349
- Astellas. XOSPATA Support Solutions. https://www.astellaspharmasupportsolutions.com/patient/xospata