Last reviewed 2026-05-13 · How we verify

Xospata (GILTERITINIB)

Xospata works by blocking the activity of a specific enzyme called Leukocyte tyrosine kinase receptor.

Xospata (Gilteritinib) is a small molecule modality developed by Astellas, targeting the Leukocyte tyrosine kinase receptor. It was FDA-approved in 2018 for the treatment of Acute Myeloid Leukemia. Xospata is a patented medication with no generic manufacturers available. Key safety considerations include its potential effects on the liver and bone marrow. As a targeted therapy, Xospata works by inhibiting the activity of a specific enzyme involved in cancer cell growth.

At a glance

Mechanism of action

Gilteritinib is small molecule that inhibits multiple receptor tyrosine kinases, including FMS-like tyrosine kinase (FLT3). Gilteritinib demonstrated the ability to inhibit FLT3 receptor signaling and proliferation in cells exogenously expressing FLT3 including FLT3-ITD, tyrosine kinase domain mutations (TKD) FLT3-D835Y and FLT3-ITD-D835Y, and it induced apoptosis in leukemic cells expressing FLT3-ITD.

Approved indications

• Acute myeloid leukemia, disease

Boxed warnings

• WARNING: DIFFERENTIATION SYNDROME Patients treated with XOSPATA have experienced symptoms of differentiation syndrome, which can be fatal or life-threatening if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, or renal dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution [see Warnings and Precautions ( 5.1 ) and Adverse Reactions ( 6.1 )] . WARNING: DIFFERENTIATION SYNDROME See full prescribing information for complete boxed warning. Patients treated with XOSPATA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution. ( 5.1 , 6.1 )

Common side effects

• Transaminase increased
• Myalgia/arthralgia
• Fatigue/malaise
• Fever
• Mucositis
• Edema
• Rash
• Noninfectious diarrhea
• Dyspnea
• Nausea
• Cough
• Constipation

Key clinical trials

• Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial) (PHASE2)
• MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
• A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML) (PHASE1)
• VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML (PHASE3)
• A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1) (PHASE1,PHASE2)
• MRD-guided Maintenance Post-HCT: Gilteritini vs Sorafenib (PHASE3)
• Study of NMS-03592088 in Patients With Relapsed or Refractory AML or CMML (PHASE1,PHASE2)
• SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation (PHASE1)

Patents

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Xospata CI brief — competitive landscape report
• Xospata updates RSS · CI watch RSS
• Astellas Pharma portfolio CI