XOSPATA Is the First Oral Monotherapy to Deliver Superior Overall Survival vs
Salvage Chemotherapy in Relapsed or Refractory FLT3m+ AML2*
Final Analysis Final Analysis
Select Safety Information

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.

*FLT3 mutation status: FLT3-ITD, FLT3-TKD, and FLT3-ITD-TKD.2

The OS endpoint was measured from the date of randomization until death by any cause in the final analysis, which included 371 patients randomized 2:1 to receive XOSPATA or a prespecified salvage chemotherapy regimen.2

CR defined as normal marrow differential with <5% blasts, ANC ≥1.0 x 109/L and platelets ≥100 x 109/L, no evidence of extramedullary leukemia, and must have been RBC and platelet transfusion independent.2

§CRh defined as marrow blasts <5%, partial hematologic recovery, ANC ≥0.5 x 109/L and platelets ≥50 x 109/L, no evidence of extramedullary leukemia, and could not have been classified as CR.2

AML=acute myeloid leukemia; ANC=absolute neutrophil count; CI=confidence interval; CR=complete remission; CRh=complete remission with partial hematologic recovery; FLT3=FMS-like tyrosine kinase 3; HR=hazard ratio; ITD=internal tandem duplication; m+=mutation-positive; NCCN=National Comprehensive Cancer Network; OS=overall survival; RBC=red blood cell; TKD=tyrosine kinase domain.


  1. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia V2.2020. © National Comprehensive Cancer Network, Inc. 2019. All rights reserved. Accessed 09-05-2019. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
  2. XOSPATA [package insert]. Northbrook, IL: Astellas Pharma US, Inc.