Rydapt
Rydapt (midostaurin) is a first-choice oral medication for acute myeloid leukemia (AML) that's FLT3-positive. It's taken in combination with chemotherapy for people who are newly diagnosed with this type of AML. Rydapt (midostaurin) can also help treat some types of systemic mastocytosis, a condition where there's an overgrowth of certain cells of the immune system. Some common side effects of this medication include nausea, vomiting, and high blood sugar.
What is Rydapt (midostaurin)?
What is Rydapt (midostaurin) used for?
- Acute myeloid leukemia (AML) with a specific mutation (FLT3-positive)
- Mast cell leukemia and systemic mastocytosis
How Rydapt (midostaurin) works
Rydapt (midostaurin) is a type of targeted therapy called a tyrosine kinase inhibitor. Kinases are enzymes (proteins) that send signals to cells, including cancer cells, that tells them to grow. Rydapt (midostaurin) works by blocking several kinases in blood cancer (leukemia) cells, which then prevents these cells from growing. Rydapt (midostaurin) works specifically on leukemia cells that have the FLT3 mutation.
For systemic mastocytosis and mast cell leukemia, Rydapt (midostaurin) is thought to work by slowing or stopping the growth of abnormal mast cells. It might also block the release of histamines that cause symptoms of systemic mastocytosis.
Drug facts
Side effects of Rydapt (midostaurin)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
Please note: The side effects listed below includes those reported by people who took Rydapt (midostaurin) for AML and systemic mastocytosis.
- Nausea (up to 83%)
- Fever from low white blood cell counts (up to 83%)
- High blood sugar levels (up to 80%)
- Lower blood calcium levels (up to 74%)
- Vomiting (up to 68%)
- Redness, pain, or ulcers on the inside of the mouth (up to 66%)
- Abnormal blood cell counts (up to 66%)
- Diarrhea (54%)
- Headache (46%)
- Swelling in the hands and feet (40%)
- Abnormal liver labs (up to 39%)
- Higher pancreatic enzyme levels (up to 37%)
- Higher uric acid levels (up to 37%)
- Red/brown/purple spots on skin (36%)
Less Common Side Effects
- Stomach pain
- Stomach bleeding
- Constipation
- Hemorrhoids
- Joint stiffness
- Cough
- Trouble breathing
- Nosebleeds
- Rash
- Infections
- Sweating
- Tiredness
- Muscle or bone pain
- Trouble sleeping
Rydapt (midostaurin) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Lung problems: cough, trouble breathing, chest pain
The following Rydapt (midostaurin) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions or angioedema—skin rash, itching or hives, swelling of the face, eyes, lips, tongue, arms, or legs, trouble swallowing or breathing
- Dry cough, shortness of breath or trouble breathing
- Infection—fever, chills, cough, sore throat, wounds that don't heal, pain or trouble when passing urine, general feeling of discomfort or being unwell
- Low red blood cell level—unusual weakness or fatigue, dizziness, headache, trouble breathing
- Unusual bruising or bleeding
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
- Bone, joint, or muscle pain
- Constipation
- Headache
- Nausea
- Pain, redness, or swelling with sores inside the mouth or throat
- Swelling of the ankles, hands, or feet
- Vomiting
Pros and cons of Rydapt (midostaurin)
Pros
- Taken by mouth
- First-choice treatment option for newly diagnosed FLT3-positive AML
- More precise in the way it targets cancer cells, compared to traditional chemotherapy
Cons
- Taken twice daily
- Must take with food
- Commonly causes nausea and vomiting
Pharmacist tips for Rydapt (midostaurin)
- Take Rydapt (midostaurin) with food. Take the medication twice a day, about every 12 hours, and swallow the capsule whole. Don’t chew, crush, or break open the capsule.
- Rydapt (midostaurin) should be avoided during pregnancy because it can harm the unborn baby. If you're female and able to become pregnant, your provider will ask you to take a pregnancy test a week before you start Rydapt (midostaurin). You should also take birth control while receiving Rydapt (midostaurin) treatment and for 4 months after your last dose.
- If you're male and have a female partner(s) who's able to become pregnant, you should also use birth control during treatment with Rydapt (midostaurin) and for 4 months after the last dose.
- If you experience any breathing problems while taking Rydapt (midostaurin), let your provider know. This medication can cause serious lung problems.
- Rydapt (midostaurin) can raise your blood sugar levels. If you already have diabetes, your provider might ask you to check your blood sugar more often to see if it’s within a healthy range. Make sure to let your provider know if you have a hard time controlling your blood sugar levels because your provider might need to make changes to your blood sugar medications.
- Let your provider know if you experience nausea or vomiting while taking Rydapt (midostaurin). If appropriate, they might prescribe medications to ease your symptoms. Your provider might also recommend other tips to prevent nausea and vomiting, such as eating bland foods, eating smaller meals more often, or drinking small sips of water throughout the day.
- If you vomit after taking Rydapt (midostaurin) (or if you miss a dose of Rydapt (midostaurin)), don’t take an extra dose to make it up. Just skip the missed dose and take your next dose of Rydapt (midostaurin) at the next scheduled time.
- Ask your provider or pharmacist about the best way to handle and throw away Rydapt (midostaurin) safely. Anticancer medications can be very harmful to people who handle or come into contact with them.
Common questions about Rydapt (midostaurin)
Rydapt (midostaurin) isn't a chemotherapy medication, it's a type of targeted therapy. Chemotherapy is a class of medication that kills cancer cells in your body. Rydapt (midostaurin) doesn’t kill cancer cells. Instead, it works to block the uncontrolled growth of cancer cells. Rydapt (midostaurin) is given in combination with chemotherapy, specifically cytarabine and daunorubicin, to treat FLT3-positive AML. Combining forces of targeted therapy and chemotherapy together allows for cancer to be treated more effectively than either one alone.
No. Although there are different types of acute myeloid leukemia (AML), Rydapt (midostaurin) is only FDA-approved to treat AML that has a change (mutation) in the FLT3 gene. This is also known as FLT3-positive AML. Your healthcare provider will perform a test to determine your FLT3 status to see if Rydapt (midostaurin) might be a treatment option for you. Knowing what type of mutations your cancer has will help you provider decide what treatment is best for you.
Nausea and vomiting are very common side effects of Rydapt (midostaurin). Because of this, you and your provider should have a plan on how to manage these side effects before you start treatment. Your provider can recommend tips, such as avoiding strong-smelling foods and eating smaller meals more often throughout the day, to ease your symptoms. Your provider might also prescribe anti-nausea medications, such as ondansetron (Zofran) or promethazine (Phenergan), for you to take during Rydapt (midostaurin) treatment. If you do experience nausea or vomiting and the symptoms don’t go away within 24 hours or gets worse, let your healthcare provider know. Your provider might have you stop treatment temporarily and possibly lower your dose of Rydapt (midostaurin).
Possibly. In animal studies, Rydapt (midostaurin) affected the ability for males to father children. Rydapt (midostaurin) also affected the ability for females to become pregnant. Talk with your provider if you've questions or concerns about how Rydapt (midostaurin) will impact fertility.
You're recommended to avoid breastfeeding while taking Rydapt (midostaurin). Animal studies show that this medication can pass into breast milk. Because of the risk of harm to nursing babies, you shouldn’t breastfeed while taking Rydapt (midostaurin) and for 4 months after your last dose. Talk with your provider about other ways to feed your baby during treatment.
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Risks and warnings for Rydapt (midostaurin)
Rydapt (midostaurin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Lung problems
It's not common, but Rydapt (midostaurin) can cause serious lung problems, like inflammation of your lungs or interstitial lung disease. Let your provider if you develop any new or worsening breathing problems. Your provider might tell you to stop taking Rydapt (midostaurin).
Harm to an unborn baby
Based on animal studies, Rydapt (midostaurin) can harm an unborn baby if taken by mothers during pregnancy. Because of this, Rydapt (midostaurin) shouldn’t be taken during pregnancy. If you’re female and able to become pregnant, your provider will ask you to do a pregnancy test a week before you start this medication. You should take birth control while taking Rydapt (midostaurin) and for at least 4 months after the last dose. Males with female partners who can become pregnant should also use birth control during treatment with Rydapt (midostaurin) and for 4 months after the last dose. Let your provider know right away if you become pregnant during this time.
Rydapt (midostaurin) dosage
Typical dosage for Rydapt (midostaurin)
- AML: The typical dosing is 50 mg by mouth twice daily on Day 8 through 21 of each treatment cycle.
- Mast cell leukemia and systemic mastocytosis: The typical dosing is 100 mg by mouth twice daily.
Your provider might adjust your dose if you experience certain side effects or if you’re taking medications that might interact with Rydapt (midostaurin).
