How Does XOSPATA Work?

FLT3 Mutations Cause Leukemia Cells to Multiply Quickly

In FLT3m+ AML, the number of leukemia cells increases. This leaves less room in the bone marrow for healthy blood cells to develop.

Bone marrow
Healthy Bone Marrow
Healthy cell
Bone Marrow With AML
Leukemia cell with FLT3 mutation

XOSPATA Targets the Growth of FLT3m+ AML Cells*

XOSPATA is an oral prescription medicine used to treat people with FLT3m+ AML.

Bone marrow
XOSPATA Targets FLT3m+ AML cells
This may decrease the number of leukemia cells.
Healthy Blood Cells May Develop Again
With fewer leukemia cells, the bone marrow may have more room for healthy blood cells.

*This is how XOSPATA was shown to work in laboratory studies.

XOSPATA targets FLT3m+ AML cells and may cause them to no longer grow in the bone marrow.
Select Safety Information

Posterior Reversible Encephalopathy Syndrome (PRES). If you take XOSPATA, you may be at risk of developing a condition involving the brain called PRES. Tell your healthcare provider right away if you have a seizure or quickly worsening symptoms such as headache, decreased alertness, confusion, reduced eyesight, blurred vision or other visual problems. Your healthcare provider will do a test to check for PRES. Your healthcare provider will stop XOSPATA if you develop PRES.

How XOSPATA May Help

XOSPATA may help those with relapsed or refractory FLT3m+ AML achieve remission and may reduce the need for blood transfusions.

How Doctors Know if You Are Responding to Treatment

In the treatment of FLT3m+ AML, doctors use multiple measures to know if people respond to treatment, including those listed below.

Helpful terms to understand
  • CR—Complete Remission
    This means the number of leukemia cells has gone down and blood counts have returned to normal.
  • CRh—Complete Remission With Partial Hematologic Recovery
    This means the number of leukemia cells has gone down, but not all healthy cells have come back.
  • Response to treatment may also be measured by how many people need blood transfusions.
 
Tap or mouse over terms in magenta to view the definition.
CR, CRh, and need for blood transfusions are all used to see if people with relapsed or refractory FLT3m+ AML like you respond to treatment.
Select Safety Information

Heart rhythm problems (QT prolongation). XOSPATA may cause a heart problem called QT prolongation. Your healthcare provider should check the electrical activity of your heart with a test called electrocardiogram (ECG) before you start taking XOSPATA and during your treatment with XOSPATA. Tell your healthcare provider right away if you have a change in your heartbeat, or if you feel dizzy, lightheaded, or faint. The risk of QT prolongation is higher in people with low blood magnesium or low blood potassium levels. Your healthcare provider will do blood tests to check your potassium and magnesium levels before and during your treatment with XOSPATA.


XOSPATA Helped Some People Achieve Remission

To study the effectiveness of XOSPATA, 138 people with FLT3m+ AML, as determined by a bone marrow or blood test, who had relapsed or were refractory to treatment took a 120 mg daily dose of XOSPATA.

Approximately 1 out of 5 people had a response with XOSPATA
  • 21% of people had a response with XOSPATA
    • 12% achieved complete remission
    • 9% achieved complete remission with partial hematologic recovery

XOSPATA Helped Some People Stay in Remission

  • People who had a complete remission continued to respond for a median of 9 months
  • People who had a complete remission with partial hematologic recovery continued to respond for a median of 3 months
 
Tap or mouse over terms in magenta to view the definition.

XOSPATA Reduced the Need for Blood Transfusions in Some People

For up to 8 weeks during treatment with XOSPATA:

Nearly 1 out of 3 people who needed blood and/or platelet transfusions at the start of the study no longer needed them (33 out of 106 people)
More than half of those who were not receiving blood and/or platelet transfusions when they began treatment with XOSPATA remained transfusion-free (17 out of 32 people)
XOSPATA was shown to help some people achieve complete remission and helped some people become blood transfusion–free for up to 8 weeks.
Select Safety Information

Inflammation of the pancreas (pancreatitis). Tell your healthcare provider right away if you have severe stomach (abdomen) pain that does not go away. This pain may happen with or without nausea and vomiting.