If you have a certain form of leukemia or mastocytosis, your doctor may recommend Rydapt.

It’s a prescription drug that’s used in adults to treat these two conditions:

  • A form of newly diagnosed acute myeloid leukemia (AML), which is a type of blood cancer.
  • Certain types of mastocytosis, which is a condition that affects blood cells called mast cells.

For more details about Rydapt’s uses, see the “Is Rydapt used for AML?” and “Is Rydapt used for other conditions?” sections below.

Rydapt basics

Rydapt is a prescription medication that contains the active drug midostaurin. It belongs to a group of medications called kinase inhibitors.

Rydapt comes as capsules that you’ll swallow.

There’s no generic form available for Rydapt. Instead, it’s only available as a brand-name drug.

In this article, we describe Rydapt’s side effects, uses, dosage, and more.

Like most drugs, Rydapt may cause mild or serious side effects. The lists below describe some of the more common side effects that Rydapt may cause. These lists don’t include all possible side effects.

Keep in mind that side effects of a drug can depend on:

  • your age
  • other health conditions you have
  • other medications you may be taking

Rydapt’s side effects may differ depending on the condition that you’re taking it to treat.

Your doctor or pharmacist can tell you more about the potential side effects of Rydapt. They can also suggest ways to help reduce side effects.

Mild side effects

Here’s a short list of some of the mild side effects that Rydapt can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Rydapt’s patient information.

Mild side effects of Rydapt that have been reported include:

Mild side effects of many drugs may go away within a few days or a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.

* For more information on this side effect, see the “Side effect focus” section below.

Serious side effects

Serious side effects from Rydapt can occur, but they aren’t common. If you have serious side effects from Rydapt, call your doctor right away. But if you think you’re having a medical emergency, you should call 911 or your local emergency number.

Serious side effects of Rydapt that have been reported include:

* For more information on this side effect, see the “Side effect focus” section below.

Side effect focus

Learn more about some of the side effects Rydapt may cause.

Severe lung problems

Taking Rydapt may increase your risk for serious lung conditions. For instance, you may develop pneumonitis or interstitial lung disease. These conditions involve inflammation of your lungs.

Normally, when you breathe in, tiny blood vessels in your lungs carry oxygen into your bloodstream. Then, when you breathe out, carbon dioxide that’s carried in your bloodstream to your lungs is exhaled.

But with inflammation in your lungs, this air exchange process doesn’t work as well as usual. So, you may have the following symptoms:

Rarely, some people taking Rydapt have had serious lung problems, some of which led to death. Lung problems may develop in people taking Rydapt with or without chemotherapy drugs.

What might help

If you develop a cough, chest pain, or trouble breathing, you shouldn’t wait to see if it goes away before telling your doctor. Instead, you must get medical attention right away.

If you develop a serious lung problem with Rydapt, your doctor will have you stop taking the drug. And you’ll most likely receive treatment in a hospital for your lungs.

Nausea

In studies, nausea was one of the most common side effects in people who took Rydapt.

Nausea from Rydapt is usually mild and temporary. But for some people, nausea and vomiting from Rydapt may be severe.

What might help

Your doctor will prescribe one or more nausea medications for you along with Rydapt. These medications help to prevent or treat nausea from Rydapt.

Some examples of medications prescribed to help prevent nausea from Rydapt include:

  • aprepitant (Emend)
  • ondansetron (Zofran)
  • granisetron (Sancuso)
  • palonosetron (Aloxi)
  • dexamethasone

You’ll take your nausea medication before you take each dose of Rydapt. For details about this, follow the directions on your prescriptions, or ask your doctor or pharmacist.

In addition to taking your prescribed nausea medications, here are some additional tips that may help ease nausea:

  • Eat small, frequent meals instead of three large meals each day.
  • Avoid eating foods that are spicy, greasy, or have a strong odor.
  • Try sipping on ginger ale that’s flat or tea that contains ginger.
  • Increase airflow around you by using a fan, opening a window, or going outdoors.
  • Consider meditation or other relaxation techniques.

If nausea from Rydapt is bothersome or becomes severe for you, talk with your doctor.

Allergic reaction

Some people may have an allergic reaction to Rydapt. Although it wasn’t common, severe allergic reaction did occur when Rydapt was used for systemic mastocytosis in a study. (For information about systemic mastocytosis, see the “Is Rydapt used for other conditions?” section below.)

Symptoms of a mild allergic reaction can include:

  • rash
  • itchiness
  • flushing (temporary warmth, redness, or deepening of skin color)

A more severe allergic reaction is rare but possible. Symptoms can include swelling under the skin, typically in your eyelids, lips, hands, or feet. They can also include swelling of your tongue, mouth, or throat, which can cause trouble breathing.

In a study, one person taking Rydapt for systemic mastocytosis had a severe allergic reaction. Their symptoms included trouble breathing and swelling under their skin.

Call your doctor right away if you have an allergic reaction to Rydapt. But if you think you’re having a medical emergency, call 911 or your local emergency number.

Your doctor will explain how you should take Rydapt. They will also explain how much to take and how often. Be sure to follow your doctor’s instructions. Below are commonly used dosages, but always take the dosage your doctor prescribes.

Taking Rydapt

Rydapt comes as capsules that you’ll swallow. It’s available in one strength: 25 milligrams (mg).

Dosage

You should take your Rydapt doses twice each day, about 12 hours apart. For example, you may take your first dose at 7:00 a.m. and your second dose at 7:00 p.m.

Rydapt’s typical dosing schedule and the number of capsules that you’ll take depend on your condition.

For instance, if you have:

For information about AML and mastocytosis, see the “Is Rydapt used for AML?” and “Is Rydapt used for other conditions?” sections below. Be sure to talk with your doctor about your prescribed dosage and treatment schedule as well.

Taking Rydapt with other drugs

When your doctor prescribes Rydapt for you, they’ll also prescribe one or more nausea medications for you.

Some examples of medications prescribed to help prevent nausea from Rydapt include:

  • aprepitant (Emend)
  • ondansetron (Zofran)
  • granisetron (Sancuso)
  • palonosetron (Aloxi)
  • dexamethasone

You’ll take the nausea medication before you take each dose of Rydapt, according to your doctor’s instructions.

This is because nausea is one of the most common side effects of Rydapt. For more details about Rydapt’s side effects, see the “What are Rydapt’s side effects?” section above.

Additionally, if you’re taking Rydapt for AML, your doctor will prescribe one or two chemotherapy drugs: cytarabine and daunorubicin.

You’ll take Rydapt as part of a treatment plan with these chemotherapy drugs. Rydapt isn’t meant to be used alone to treat AML. To learn more about AML and how Rydapt treats it, see the “Is Rydapt used for AML?” section below.

Questions about taking Rydapt

Here’s a list of answers to some common questions about taking Rydapt.

  • What if I miss a dose of Rydapt? If you miss a dose of Rydapt, just take your next dose at the regular time. Don’t take an extra dose or take your next dose early. Also, if you vomit after taking Rydapt, don’t take another dose of the drug. Doing so could increase your risk for serious side effects.
  • Can Rydapt capsules be opened, crushed, or cut? No, Rydapt capsules should be swallowed whole. Don’t open, crush, cut, or chew the capsules.
  • Should I take Rydapt with food? Yes, you should take Rydapt with food.
  • How long does Rydapt take to work? Rydapt begins working soon after you start taking it. In studies, some people with mastocytosis had a response to treatment within 2 weeks. Talk with your doctor about how you’ll be monitored for your body’s response to treatment.
Questions for your doctor

You may have questions about Rydapt and your treatment plan. It’s important to discuss all your concerns with your doctor.

Here are a few tips that might help guide your discussion:

  • Before your appointment, write down questions like:
    • How will Rydapt affect my body, mood, or lifestyle?
  • Bring someone with you to your appointment if doing so will help you feel more comfortable.
  • If you don’t understand something related to your condition or treatment, ask your doctor to explain it to you.

Remember, your doctor and other healthcare professionals are available to help you. And they want you to get the best care possible. So, don’t be afraid to ask questions or offer feedback on your treatment.

Costs of prescription drugs can vary depending on many factors. These factors include what your insurance plan covers and which pharmacy you use.

If you have questions about how to pay for your prescription, talk with your doctor or pharmacist. You can also visit the Rydapt manufacturer’s website to see if they have support options.

Rydapt is used to treat two types of conditions in adults, one of which is acute myeloid leukemia (AML). The other condition, called mastocytosis, is described in the “Is Rydapt used for other conditions?” section, just below.

For AML, Rydapt is prescribed in combination with one or two chemotherapy drugs: cytarabine and daunorubicin. Your doctor will prescribe one or both of these drugs together with Rydapt as part of your AML treatment plan. Rydapt isn’t meant to be used alone to treat AML.

AML treatment with Rydapt has two phases:

  • Induction phase. The induction phase is the first phase of treatment. For this phase, your doctor will prescribe Rydapt with both cytarabine and daunorubicin. This three-drug combination is designed to kill as many cancer cells in your body as possible.
  • Consolidation phase. The consolidation phase is the second phase of treatment. For this second phase, your doctor will prescribe Rydapt with cytarabine. This two-drug combination is meant to kill any cancer cells that survived after the induction phase.

What AML is and what Rydapt does for it

AML is a cancer that affects your blood and bone marrow. Specifically, AML affects your white blood cells and causes them to form more quickly than usual.

Rydapt is used to treat AML 迟丑补迟’蝉:

  • newly diagnosed, and
  • positive for a mutated (abnormal) FLT3 gene (FLT3-positive)

If you have FLT3-positive AML, your body makes enzymes called kinases. These enzymes are proteins that help cancer cells grow and spread.

Rydapt belongs to a group of drugs known as kinase inhibitors. Rydapt, like other kinase inhibitors, is a type of targeted therapy. These drugs work on specific targets in your body.

Rydapt works by inhibiting (blocking) the actions of specific kinases, including FLT3. This action stops certain cancer cells from growing and spreading.

Note: Rydapt has also been used off-label in children with AML. (Off-label drug use is when a drug is prescribed for a condition other than its approved use.) But, Rydapt is currently only approved for use in adults. Its safety and effectiveness in children aren’t known.

Rydapt is used to treat two types of conditions in adults, one of which is acute myeloid leukemia (AML). It’s described in the “Is Rydapt used for AML?” section, just above.

The other condition Rydapt is used to treat is mastocytosis, which is a condition that affects mast cells. Mast cells are a type of white blood cell that normally help your body fight off infections.

With mastocytosis, your body makes too many mast cells. These cells can build up in your body. This buildup can interfere with your body’s ability to make healthy blood cells. In some people, mastocytosis can lead to cancer.

Specifically, Rydapt is used to treat three forms of mastocytosis in adults:

  • Aggressive systemic mastocytosis. This form of mastocytosis causes abnormal mast cells to form clusters in your bone marrow and organs, including your liver and spleen.
  • Systemic mastocytosis with associated hematological neoplasm. With this condition, systemic mastocytosis results from a hematological neoplasm. (Hematological neoplasm is a general term that refers to cancers in the blood.) With this condition, other blood cell types are affected along with mast cells.
  • Mast cell leukemia (MCL). MCL is a rare form of leukemia (a type of cancer) that can develop in people with aggressive systemic mastocytosis. With MCL, high levels of mast cells build up in your blood and bone marrow.

Rydapt belongs to a group of drugs known as kinase inhibitors. Rydapt, like other kinase inhibitors, is a type of targeted therapy. These drugs work on specific targets in your body.

Rydapt works by slowing or stopping how quickly mast cells are made in your body. It also slows or stops their action. The drug is also thought to block histamine, which can cause symptoms of mastocytosis. Histamine is a chemical released by mast cells that can cause inflammation. Its release can be triggered by certain health conditions or during an allergic reaction.

Get answers to some commonly asked questions about Rydapt.

Is Rydapt a chemotherapy drug?

No, Rydapt it isn’t a chemotherapy drug. But it is used to treat certain types of cancer, including acute myeloid leukemia.

Chemotherapy drugs work by killing cells that grow and divide quickly. This includes cancer cells. But chemotherapy drugs don’t only kill cancer cells. They can also kill other cells that grow and divide quickly, including healthy cells.

This non-specific action of chemotherapy is why some chemotherapy drugs cause many side effects.

Rydapt is a type of targeted therapy called a kinase inhibitor. These drugs work on specific targets in your body.

Rydapt blocks a certain kinase enzyme (a type of protein). This blocking action stops certain cancer cells from growing and spreading.

Will I need to use Rydapt long term?

Your length of treatment with Rydapt depends on the condition you’re taking it to treat.

If you have acute myeloid leukemia, you won’t take Rydapt long term. Instead, you’ll take it for up to six cycles of treatment. (And it’s taken in combination with chemotherapy.) Each treatment cycle is usually 21 days long.

If you have mastocytosis, you may take Rydapt long term. But that depends on how well the drug is working for you, and whether you have serious or bothersome side effects. Your doctor will monitor how well Rydapt is working, and they’ll check for certain side effects by having your blood tested regularly.

If you have more questions about how long you may need to take Rydapt, talk with your doctor.

There are some important things to talk about with your doctor when considering Rydapt treatment. For instance, be sure to discuss:

  • your overall health
  • any current or past medical conditions
  • any medications, herbs, or supplements that you take

Your doctor will also order certain blood tests for you before you start taking Rydapt. These tests are used to check your blood cell levels and the health of your kidneys and liver.

Interactions

Taking certain drugs, vaccines, foods, and supplements can affect how medications work. These effects are called interactions.

Before taking Rydapt, be sure to tell your doctor about all medications you take (including prescription and over-the-counter types). Also describe any vitamins, herbs, or supplements you use. Your doctor or pharmacist can tell you about any interactions these items may cause with Rydapt.

Interactions with drugs or supplements

Rydapt can interact with several types of drugs. These drugs include certain:

This list does not contain all types of drugs that may interact with Rydapt. Your doctor or pharmacist can tell you more about these interactions and any others that may occur with use of Rydapt.

Also, you shouldn’t take St. John’s wort while you’re taking Rydapt. St. John’s wort is an herb that’s sometimes used as a natural remedy for depression. But St. John’s wort can lower the levels of Rydapt in your body, making it less effective for you.

Rydapt may also interact with other herbal supplements. So, it’s important to tell your doctor and pharmacist about everything that you take, including any herbs and supplements.

Other interactions

It’s possible that Rydapt can interact with certain foods.

For example, you shouldn’t eat grapefruit or drink grapefruit juice while you’re taking Rydapt. Grapefruit is known to raise the levels of certain drugs in your body, including Rydapt. This interaction can increase your risk for serious side effects from the drug.

Warnings

Rydapt may not be right for you if you have certain medical conditions or other factors that affect your health. Talk with your doctor about your health history before you take Rydapt. Factors to consider include:

  • Allergic reaction. If you’ve had an allergic reaction to Rydapt or any of its ingredients, you shouldn’t take Rydapt. Ask your doctor about other medications that may be better options for you.

Use with alcohol

It’s best to limit the amount of alcohol you drink while you’re taking Rydapt.

There’s no harmful interaction between Rydapt and alcohol. But, consuming too much alcohol can cause some of the same side effects that Rydapt causes. These include:

  • nausea
  • vomiting
  • diarrhea
  • headache
  • fatigue (lack of energy)

If you drink alcohol, talk with your doctor about how much is safe for you to drink while taking Rydapt.

Pregnancy and breastfeeding

Rydapt isn’t recommended for use during pregnancy. This is because there’s not enough information available to know if this drug is safe to take during pregnancy.

If you’re a female* who can become pregnant, your doctor will check to make sure you have a negative pregnancy test within 7 days before starting Rydapt. And they’ll recommend that birth control be used by both males* and females taking this drug.

If you become pregnant while you or your partner is taking Rydapt, tell your doctor right away. If you and your doctor decide that you’ll continue with Rydapt treatment during the pregnancy, consider enrolling in Rydapt’s pregnancy registry. (Pregnancy registries collect information about a drug’s use during pregnancy.) To learn more about this drug registry, call 888-669-6682 or visit this website.

Additionally, it’s best to avoid breastfeeding while you’re taking Rydapt. The drug could pass into breast milk and cause harm to a child who is breastfed. You should wait at least 4 months after your last dose of Rydapt before breastfeeding.

If you’re pregnant or breastfeeding, talk with your doctor before taking Rydapt. They’ll recommend the best treatment plan for you during this time.

* In this article, we use the terms “male” and “female” to refer to someone’s sex assigned at birth. For information about the difference between sex and gender, see this article.

Don’t take more Rydapt than your doctor prescribes. Taking more than your recommended dose of this medication could increase your risk for serious side effects.

What to do in case you take too much Rydapt

Call your doctor if you think you’ve taken too much Rydapt. You can also call 800-222-1222 to reach the American Association of Poison Control Centers, or use its online resource. But if you have severe symptoms, immediately call 911 (or your local emergency number) or go to the nearest emergency room.

If you’ve been diagnosed with acute myeloid leukemia (AML) or a certain type of mastocytosis, your doctor may prescribe Rydapt.

For more information about treatment outcomes with AML, read this article. You may also want to see this overview of treatment options for AML.

If you have questions about Rydapt, talk with your doctor or pharmacist. If you’re not sure what to ask, here’s a list of questions to consider:

  • Should I keep taking my other medications while I’m taking Rydapt?
  • Will I have to take Rydapt for the rest of my life if I have mastocytosis?
  • Do my medical conditions put me at a higher risk for side effects with Rydapt?
  • Can I use herbal remedies to help ease side effects of Rydapt?

Q:

Is Rydapt the only treatment option available for FLT3-positive acute myeloid leukemia (AML)?

Anonymous patient

A:

No, it’s not.

Rydapt is used to treat AML 迟丑补迟’蝉:

  • newly diagnosed, and
  • positive for a mutated (abnormal) FLT3 gene (FLT3-positive)

Other treatments have been studied for FLT3-positive AML. But the only other treatment that’s approved for FLT3-positive AML is gilteritinib (Xospata).

Unlike Rydapt, which is approved to treat newly diagnosed AML that’s FLT3-positive, Xospata is only approved to treat FLT3-positive AML that’s been treated in the past.

You can read more about Xospata here. And if you have questions about which treatment is right for you, ask your doctor.

Melissa Badowski, PharmD, MPH, FCCPAnswers represent the opinions of our medical experts. All content is strictly informational and should not be considered medical advice.

Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up-to-date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or other healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.