OnCo
drugsProductApproved2018🇪🇺🇬🇧🇯🇵🇨🇳🇦🇺

Gilteritinib

A single pill that beats salvage chemotherapy for relapsed FLT3-mutated AML, and the backbone of the triplets now being tested in frontline disease.

ADMIRAL (n=371): gilteritinib vs salvage chemotherapy in relapsed/refractory FLT3-mutated AML, OS 9.3 vs 5.6 months (HR 0.64), CR/CRh 34%. Approved 2018. Post-transplant maintenance (MORPHO) reduced relapse in MRD-positive patients. Frontline triplets (gilteritinib + azacitidine + venetoclax) show high CR rates in unfit patients; the phase 3 vs midostaurin (HOVON 156 AML) reported non-superiority for EFS in 2025-26 data, keeping midostaurin/quizartinib as intensive standards.

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Gilteritinib
PubChem
Mechanism, step by step
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1.Gilteritinib binds active FLT3 regardless of ITD or TKD mutation

Modality
Small-molecule kinase inhibitor (FLT3, type I)
Mechanism
Type I FLT3/AXL inhibitor active against ITD and TKD (D835) mutations.
Brand / code
Xospata
Dosing & schedule
Route
Oral
Schedule
120 mg once daily until progression or unacceptable toxicity (minimum 6 months to allow response)
Monitoring
Differentiation syndrome (boxed warning), QT, LFTs, PRES, pancreatitis

Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.

Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).

Commercial insurance
covered with prior authorisation

Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. FLT3 mutation by an approved test.

Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

NICE recommendedNICE TA642 · 2020SMC: accepted
Appraised for
Relapsed or refractory FLT3 mutation-positive AML
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE TA642 · SMC advice: gilteritinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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  1. 28 Nov 2018ApprovalUS

    ADMIRAL interim; full OS data 2019

Approvals

RegionYearIndication
US2018Relapsed or refractory FLT3-mutated AML

Safety

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Toxicity profile
Adverse eventAny gradeGrade 3+
Differentiation syndrome
3%
Transaminase elevation
44%
Myalgia/arthralgia
42%
Febrile neutropenia
46%

Boxed warning. Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
44 studies19 recruiting33 Phase 213 Phase 3
Search “Gilteritinib” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Gilteritinib
intervention: Gilteritinib
Open on ClinicalTrials.gov →

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Landmark trials in OnCo

Key papers

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Latest papers

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Literature trend108 papers in the last 12 months+19% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Gilteritinib" OR ABSTRACT:"Gilteritinib" OR TITLE:"Xospata" OR ABSTRACT:"Xospata") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Gilteritinib, not a curated reading list.

Connected

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