Frequently Asked Questions

Acute myeloid leukemia, or AML, is a type of blood cancer that starts in the bone marrow, where blood cells are made. In FLT3m+ AML, mutations in the FLT3 gene cause abnormal (leukemia) cells to grow and multiply, leaving less room for healthy blood cells to develop.

Mutations in the FLT3 gene are the most common types of mutations in AML. About 1 in 3 people diagnosed with AML may have a FLT3 mutation.

Relapse is when AML comes back after a period of improvement with treatment. Refractory means AML has not improved after previous treatment(s).

A targeted therapy, like XOSPATA, works by targeting specific parts of cancer cells in order to lower the number of these cells in the body.

XOSPATA is a prescription medicine used to treat adults with acute myeloid leukemia (AML) with a FLT3 mutation when the disease has come back or has not improved after previous treatment(s). Your healthcare provider will perform a test to make sure that XOSPATA is right for you. It is not known if XOSPATA is safe and effective in children.

XOSPATA is a targeted therapy and works differently than traditional chemotherapy. Traditional chemotherapy works by killing fast-growing cells, which can include cancer cells and other types of cells. Targeted therapies are designed to target certain features of cancer cells, like mutations. Other cells in the body may still be affected during treatment, but this type of therapy is designed with a specific target in mind.

In FLT3m+ AML, the number of leukemia cells increases in the bone marrow, leaving less room for healthy cells to grow. XOSPATA targets the FLT3 mutation, which may cause leukemia cells to stop growing in the bone marrow.* With fewer leukemia cells, the bone marrow may have more room for healthy blood cells.

Your doctor may use different measures to know if you are responding to treatment. One way is to check your blood counts to measure the number of leukemia cells in your body. When there are no signs of leukemia cells found in the blood or bone marrow, and blood counts have returned to normal, it is called complete remission. This does not mean that the cancer has been cured.

It may take time to see a response with XOSPATA. Your doctor may treat you with XOSPATA for a minimum of 6 months, as long as your disease is not getting worse and there are no serious side effects.

The most common side effects of XOSPATA include:

• Changes in liver function tests
• Joint or muscle pain
• Tiredness
• Fever
• Pain or sores in mouth or throat
• Swelling of arms or legs
• Rash
• Diarrhea
• Shortness of breath
• Nausea
• Cough
• Constipation
• Eye problems
• Headache
• Dizziness
• Low blood pressure
• Vomiting
• Decreased urination

Your healthcare provider may tell you to decrease your dose, temporarily stop, or completely stop taking XOSPATA if you develop certain side effects during treatment with XOSPATA.

These are not all of the possible side effects of XOSPATA. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Before taking XOSPATA, tell your healthcare provider about all of your medical conditions, including if you:

• Have any heart problems, including a condition called long QT syndrome
• Have problems with abnormal electrolytes such as sodium, potassium, or magnesium levels
• Are pregnant or plan to become pregnant. XOSPATA can cause harm to your unborn baby. You should avoid becoming pregnant during treatment with XOSPATA. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with XOSPATA
  • If you are able to become pregnant, your healthcare provider may perform a pregnancy test 7 days before you start treatment with XOSPATA
  • Females who are able to become pregnant should use effective birth control (contraception) during treatment with XOSPATA and for 6 months after the last dose of XOSPATA
  • Males who have female partners that are able to become pregnant should use effective birth control (contraception) during treatment with XOSPATA and for 4 months after the last dose of XOSPATA
• Are breastfeeding or plan to breastfeed. It is not known if XOSPATA passes into your breast milk. Do not breastfeed during treatment with XOSPATA and for 2 months after the last dose of XOSPATA

• Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. XOSPATA may affect the way other medicines work and other medicines may affect how XOSPATA works.

Take XOSPATA exactly as your healthcare provider tells you to. Do not change your dose or stop taking XOSPATA without talking to your healthcare provider. Take XOSPATA 1 time a day at about the same time each day. Swallow XOSPATA tablets whole. Do not break, crush, or chew the tablet. XOSPATA can be taken with or without food.

If you miss a dose of XOSPATA, or did not take it at the usual time, take your dose as soon as possible and at least 12 hours before your next dose. Return to your normal schedule the following day. Do not take 2 doses of XOSPATA within 12 hours.

If you have further questions about your XOSPATA dose, reach out to your doctor.

XOSPATA comes in a child-resistant package. Store XOSPATA at room temperature between 68°F to 77°F (20°C to 25°C). Keep XOSPATA in the original container to protect it from light, moisture, and humidity.

Keep XOSPATA and all medicines out of the reach of children.

XOSPATA is a specialty drug, so it cannot be filled at your local pharmacy. After your doctor writes you a prescription for XOSPATA, they can send the prescription to a specialty pharmacy or to XOSPATA Support Solutions^SM.

Next, XOSPATA Support Solutions^SM or the specialty pharmacy will call you to confirm details about your address and insurance coverage.

After your XOSPATA prescription is approved by your insurance company, it will be dispensed and shipped by a XOSPATA network specialty pharmacy right to your home. You will then receive a call from the specialty pharmacy to arrange refills of your prescription.

Your doctor may also be able to provide XOSPATA in their office. Ask your doctor if this is an option for you.

If XOSPATA is not covered by your insurance, XOSPATA Support Solutions^SM may be able to help. XOSPATA Support Solutions^SM helps people who have been prescribed XOSPATA access their medication as quickly as possible.

When you call XOSPATA Support Solutions^SM, a case manager will work with you to understand your healthcare coverage and identify financial assistance information that may be available to help.

To speak with a case manager, call XOSPATA Support Solutions^SM at 1-844-632-9272, Monday through Friday, 8:30 AM to 8:00 PM ET.

If you have commercial prescription insurance, you may be eligible* for the XOSPATA Copay Card Program. With the copay card, you pay as little as $0 per prescription, up to a maximum savings of $25,000 per calendar year, and are enrolled in the program for a 12-month period. There are no income requirements. Talk to your doctor or pharmacist about enrolling in the XOSPATA Copay Card Program.