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 Was XOSPATA Studied?

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
  • OS—Overall Survival
    This refers to the length of time during a study that people stay alive beyond diagnosis or start of treatment.
  • 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 blood counts have returned to normal.

How well a treatment works may also be measured by how many people need blood transfusions.

 
Tap or mouse over terms in magenta to view the definition.

XOSPATA Was Compared to Chemotherapy

The effectiveness of XOSPATA, compared to chemotherapy, was studied in people with FLT3m+ AML, as determined by a bone marrow or blood test, who had relapsed or were refractory to treatment.

study-design

The study included people who were treated with XOSPATA or chemotherapy. Treatment results were examined at 2 different time points in the study.

First Set of Results

Included 138 people who had been treated with XOSPATA and how many of them achieved remission (CR/CRh). These results were available before the study was over.

Final Set of Results

Compared the overall survival (OS) in 247 people who had taken XOSPATA with 124 people who were treated with chemotherapy.

Select Safety Information

Changes in the electrical activity of your heart called QTc prolongation. QTc prolongation can cause irregular heartbeats that can be life-threatening. Your healthcare provider will check the electrical activity of your heart with a test called an electrocardiogram (ECG) before you start taking XOSPATA and during your treatment with XOSPATA. Tell your healthcare provider right away 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.

How XOSPATA May Help

XOSPATA is the first FDA-approved targeted therapy for people with relapsed or refractory FLT3m+ AML and may lead to a longer life.

XOSPATA Helped Some People Live Longer and Achieve Remission

The two main results in this study were OS and CR/CRh.

First Set of Results
Nearly 1 out of 5

people had a favorable response to treatment with XOSPATA

  • 12% achieved complete remission
  • 9% achieved complete remission with partial hematologic recovery

People who had a favorable response to treatment with XOSPATA in the first assessment continued to stay in remission for a median of nearly 5 months.

 
Tap or mouse over terms in magenta to view the definition.
Final Set of Results

During the study, people who took XOSPATA lived for a median of

study-design
More From the Final Set of Results

XOSPATA Helped Some People Achieve Remission

1 out of 7

people achieved complete remission

  • Out of the 247 people who took XOSPATA, 14% achieved complete remission during the study vs 11% with chemotherapy

XOSPATA Helped Some People Stay in Remission

People who had a favorable response to treatment with XOSPATA continued to stay in remission for a median of 15 months, compared to a median of 2 months with chemotherapy.


XOSPATA Reduced the Need for Blood Transfusions in Some People

For up to 8 weeks during treatment with XOSPATA:

Nearly1 out of 3 people who needed blood and/or platelet transfusions when they began treatment with XOSPATA no longer needed them (68 out of 197 people)
More than half of those who were not receiving blood and/or platelet transfusions when they began treatment with XOSPATA remained transfusion-free (29 out of 49 people)
Ask your doctor if XOSPATA is right for you.

FDA=Food and Drug Administration.

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.