Rydapt (midostaurin)

What is Rydapt?

Rydapt is a brand-name prescription medication. It’s approved to treat the following conditions in adults:

• Newly diagnosed acute myeloid leukemia (AML) in people with a certain genetic mutation. AML is a type of cancer that’s found in your blood and bone marrow. Rydapt is given to people with AML who have an abnormal (mutated) FLT3 gene. For this use, Rydapt is used in combination with chemotherapy drugs.
• Certain types of systemic mastocytosis. This condition affects certain blood cells called mast cells. In some cases, it can lead to blood cancer. Rydapt can be used alone to treat the following types of systemic mastocytosis:
  • aggressive systemic mastocytosis (ASM)
  • systemic mastocytosis that occurs with a form of cancer called associated hematological neoplasm (SM-AHN)
  • mast cell leukemia (MCL), which is a type of cancer that may develop from ASM

Rydapt comes as capsules that are taken by mouth twice daily. It contains the drug midostaurin, which belongs to a class of drugs called kinase inhibitors. (A class of drugs describes medications that work in the same way.)

Rydapt isn’t considered a chemotherapy drug. Chemotherapy refers to traditional drugs used to treat cancer. These drugs typically affect both cancer cells and normal cells in your body. Rydapt, on the other hand, is a targeted drug. It’s designed to work mainly on cancer cells.

FDA approval

In 2017, the Food and Drug Administration (FDA) approved Rydapt for use. It was the first targeted therapy drug approved to treat AML.

Effectiveness

In a clinical study, Rydapt was effective in treating AML. People in this study were given either Rydapt or a placebo (treatment with no active drug). Both groups of people were also given chemotherapy drugs. The results showed the following:

• People taking Rydapt were 23% more likely to still be alive after at least 3.5 years than were people taking the placebo.
• About 50% of the people taking Rydapt went at least 8 months without having certain complications caused by their cancer. In comparison, about 50% of people taking the placebo went at least 3 months without having certain complications caused by their cancer.

In another clinical study, Rydapt was effective in treating certain types of systemic mastocytosis. In this study, 21% of people taking Rydapt alone had complete remission* or partial remission.** These results were reported after six cycles of treatment. (The length of these treatment cycles varied.)

* With complete remission, the people had an absence of mutated (abnormal) mast cells.

** With partial remission, the people had reduced activity of mutated mast cells.

Rydapt generic

Rydapt is available only as a brand-name medication. It’s not currently available in generic form. A generic drug is an exact copy of the active drug in a brand-name medication.

Rydapt contains the active ingredient midostaurin.

Rydapt cost

As with all medications, the cost of Rydapt can vary.

The actual price you’ll pay depends on your insurance plan, your location, and the pharmacy you use.

Your insurance plan may require you to get prior authorization before approving coverage for Rydapt. This means that your doctor and insurance company will need to communicate about your prescription before the insurance company will cover the drug. The insurance company will review the request and let you and your doctor know if your plan will cover Rydapt.

If you’re not sure if you’ll need to get prior authorization for Rydapt, contact your insurance company.

Financial and insurance assistance

If you need financial support to pay for Rydapt, help is available.

Novartis Pharmaceuticals Corporation, the manufacturer of Rydapt, offers assistance through its Novartis Oncology Universal Co-Pay Program, which helps with monthly copays. For more information and to find out if you’re eligible, call 877-577-7756 or visit the program website.

A program called RYDAPT NOW is also available. This program may help make Rydapt available to you before your insurance begins to cover the medication. For more information about RYDAPT NOW, call 800-282-7630 or visit the program website. And talk with your doctor about whether you’re eligible for this support.

Generic version

Rydapt isn’t available in a generic form. (A generic drug is an exact copy of the active drug in a brand-name medication.) Generics tend to cost less than brand-name drugs.

Rydapt side effects

Rydapt can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking Rydapt. These lists don’t include all the possible side effects.

Your side effects might differ from those listed below, depending on whether you’re using Rydapt to treat either acute myeloid leukemia (AML) or systemic mastocytosis.

For more information on the possible side effects of Rydapt, talk with your doctor or pharmacist. They can give you tips on how to deal with any side effects that may be bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. If you would like to report to the FDA a side effect you’ve had with Rydapt, you can do so through MedWatch.

Mild side effects

The mild side effects of Rydapt can include:

• nausea
• vomiting
• diarrhea
• headache
• muscle or bone pain
• fatigue (lack of energy)
• belly pain
• upper respiratory infections, such as the common cold

Most of these side effects may go away within a few days or a couple of weeks. If they’re more severe or don’t go away, talk with your doctor or pharmacist.

Serious side effects

Serious side effects from Rydapt can be common. Call your doctor right away if you have serious side effects. Call 911 if your symptoms feel life threatening or if you think you’re having a medical emergency.

Serious side effects and their symptoms can include the following:

• Infections, such as urinary tract infections (UTIs), pneumonia, and herpes (after exposure to the herpes virus). Symptoms of infection will vary, but can include:
  • fever
  • malaise (generally not feeling well)
  • fatigue (lack of energy)
• Febrile neutropenia (a condition that causes both a fever and low levels of a certain type of white blood cell). In addition to fever, symptoms can include:
  • chills
  • sweating
  • sores inside your mouth
  • frequent infections
• Pneumonitis (inflammation in your lungs) and lung disease. Symptoms can include:
  • fever
  • chills
  • muscle pain
  • trouble breathing
  • dry cough
  • tight feeling in your chest
  • fatigue (lack of energy)
• Severe gastrointestinal problems. Symptoms can include:
  • nausea
  • vomiting
  • colitis (inflammation in your colon)
  • bleeding ulcers in your small intestine
  • hemorrhoids
• Severe allergic reaction, which is discussed in detail below.

Side effect details

You may wonder how often certain side effects occur with this drug. Here’s some detail on one of the side effects this drug may cause.

Allergic reaction

As with most drugs, some people can have an allergic reaction after taking Rydapt. In a clinical study, about 4% of people taking Rydapt to treat systemic mastocytosis had an allergic reaction.

Symptoms of a mild allergic reaction can include:

• skin rash
• itchiness
• flushing (warmth and redness in your skin)

A more severe allergic reaction is rare but possible. In a clinical study, one person taking Rydapt for systemic mastocytosis had a severe allergic reaction that caused trouble breathing and angioedema (swelling under the skin, typically in the eyelids, lips, hands, or feet).

In addition to trouble breathing and angioedema, symptoms of a severe allergic reaction can also include swelling in your tongue, mouth, or throat.

Call your doctor right away if you have a severe allergic reaction to Rydapt. But if your symptoms feel life threatening or if you think you’re having a medical emergency, call 911.

Rydapt dosage

The Rydapt dosage your doctor prescribes will depend on several factors. These include the condition you’re using the drug to treat and how well your body reacts to Rydapt.

Typically, your doctor will start you on the usual recommended dosage. Then they’ll adjust the dose over time to reach the amount that’s right for you. Your doctor will ultimately prescribe the smallest dosage that provides the desired effect.

The following information describes dosages that are commonly used or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to suit your needs.

Drug forms and strengths

Rydapt comes as 25-mg capsules that are taken by mouth. Rydapt should be taken with food.

Dosage for acute myeloid leukemia

The typical dosage of Rydapt for acute myeloid leukemia (AML) is 50 mg taken twice daily. For this dosage, you’ll take two 25-mg capsules twice each day.

When used to treat AML, Rydapt is given in the following two phases, which each typically last 21 days:

• Induction phase. This is the first phase of AML treatment. This phase is meant to kill as many cancer cells in your body as possible. In this phase:
  • over the first 7 days, you’ll receive two chemotherapy* drugs: cytarabine and daunorubicin
  • on days 8 through 21, you’ll receive Rydapt
• Consolidation phase. This is the second phase of AML treatment. It’s meant to treat any cancer cells that survived the induction phase of treatment. In this phase:
  • on days 1, 3, and 5, you’ll receive the chemotherapy drug cytarabine
  • on days 8 through 21, you’ll receive Rydapt

You’ll likely start the induction phase of AML treatment in a hospital. This way, your doctor will be able to oversee your dosing and monitor you for any side effects. Your dosage may need to be adjusted if you have severe side effects from Rydapt.

Usually, the consolidation phase of AML treatment can be given at an outpatient clinic or in your home. But this will depend on whether you’re having any bothersome or severe side effects during treatment. Your doctor will help you decide if this is a good option for you.

* Chemotherapy describes traditional drugs used to treat cancer.

Dosage for systemic mastocytosis

The typical dosage of Rydapt for systemic mastocytosis is 100 mg taken twice daily. For this dosage, you’ll take four 25-mg capsules twice each day.

If you have severe side effects during treatment, your doctor may prescribe a different dosage of Rydapt for you.

What if I miss a dose?

If you miss a dose of Rydapt, just take your next dose at the scheduled time. Don’t take an extra dose of Rydapt to make up for the missed dose. This will increase your risk of serious side effects from the drug.

If you vomit after taking a dose of Rydapt, don’t take another dose. Doing this can also increase your risk of serious side effects. Instead, just wait and take your next dose of Rydapt at the scheduled time.

Will I need to use this drug long term?

If you’re using Rydapt to treat AML, you won’t take the drug long term. Instead, Rydapt can be used for up to six cycles of treatment. (Each treatment cycle typically lasts for 21 days.) Specifically, Rydapt is used for two cycles of induction phase treatment and four cycles of consolidation phase treatment.

If you’re using Rydapt to treat systemic mastocytosis, you may take the drug long term if you and your doctor determine that it’s safe and effective for you. In this case, you can take Rydapt until your condition worsens or until you can no longer tolerate side effects of the drug.

Rydapt uses

The Food and Drug Administration (FDA) approves prescription drugs such as Rydapt to treat certain conditions. Rydapt is approved to treat people with the following conditions.

Rydapt for acute myeloid leukemia

Rydapt is FDA-approved to treat newly diagnosed acute myeloid leukemia (AML) in adults with a certain genetic mutation (abnormal change). It’s prescribed for people who have a mutation in their FLT3 gene. With this gene change, the AML is called FLT3-positive (FLT3+) AML.

AML is a form of cancer that affects your blood and bone marrow. Normally, your body makes healthy blood cells inside your bone marrow. But with leukemia, your body makes abnormal blood cells in your bone marrow.

As the abnormal cells begin to grow inside your bone marrow, your body can’t make as many healthy cells as usual. This leads to low levels of healthy blood cells and a buildup of abnormal blood cells.

In people with the FLT3 gene mutation, their bodies make proteins called kinases. These proteins help cancer cells grow and spread.

Rydapt works by blocking the activity of certain kinases. This helps stop the kinases from supporting cancer cell growth, which slows their growth and can cause the cancer cells to die.

When it’s used to treat AML, Rydapt is given in combination with chemotherapy drugs. (Chemotherapy describes traditional drugs used to treat cancer.) Rydapt isn’t meant to be used alone to treat AML.

Effectiveness for acute myeloid leukemia

In a clinical study, Rydapt was effective in treating AML. People in this study were given either Rydapt or a placebo (treatment with no active drug). Both groups of people were also given chemotherapy drugs. The results showed the following:

• People taking Rydapt were 23% more likely to still be alive after at least 3.5 years than were people taking the placebo.
• About 50% of the people taking Rydapt went at least 8 months without having certain complications caused by their cancer. In comparison, about 50% of people taking the placebo went at least 3 months without having certain complications caused by their cancer.

Rydapt for systemic mastocytosis

Rydapt is FDA-approved to treat systemic mastocytosis in adults. This disease affects your blood and bone marrow.

With systemic mastocytosis, your bone marrow makes abnormally high levels of mast cells (a type of white blood cell). These cells normally help your body fight off infections and heal wounds.

But when your bone marrow makes too many mast cells, they start to build up inside your body. This may lower the amount of healthy blood cells that your body is able to make. In some cases, systemic mastocytosis can lead to cancer.

Rydapt is used to treat the following three types of systemic mastocytosis:

• Aggressive systemic mastocytosis. This form of systemic mastocytosis is rare. It causes mast cells to build up in certain organs, including your liver and spleen.
• Systemic mastocytosis with associated hematological neoplasm. With this condition, systemic mastocytosis occurs with a form of cancer called hematological neoplasm. (This form of cancer affects certain types of blood cells other than mast cells.)
• Mast cell leukemia. This condition is a rare form of cancer that may develop in people with aggressive systemic mastocytosis. With mast cell leukemia, mast cells build up in your blood and bone marrow.

Rydapt can be used alone to treat systemic mastocytosis. The drug doesn’t have to be used in combination with chemotherapy for this condition.

Effectiveness for systemic mastocytosis

In a clinical study, Rydapt was effective in treating certain types of systemic mastocytosis. In this study, 21% of people taking Rydapt alone had complete remission* or partial remission.** These results were reported after six cycles of treatment. (The length of these treatment cycles varied.)

* With complete remission, the people had an absence of mutated (abnormal) mast cells.

** With partial remission, the people had reduced activity of mutated mast cells.

Rydapt use with other drugs

When used to treat acute myeloid leukemia (AML), Rydapt is used in combination with chemotherapy. (Chemotherapy describes traditional drugs used to treat cancer.)

And when it’s used to treat either AML or systemic mastocytosis, Rydapt is also given with certain medications that help prevent nausea and vomiting.

The use of Rydapt with these other drugs is described below.

Note: Some of the medications described below are used off-label. Off-label use is when a drug that’s approved to treat one condition is used for another condition.

Rydapt with chemotherapy

For AML treatment, Rydapt is given in two phases. In each phase, Rydapt is given in combination with certain chemotherapy drugs. The two phases of Rydapt treatment are as follows:

• Induction phase. This is the first phase of AML treatment. It’s meant to kill off as many cancer cells inside your body as possible. For certain portions of this phase, Rydapt is given with two chemotherapy drugs: cytarabine and daunorubicin.
• Consolidation phase. This is the second phase of AML treatment. It’s meant to treat any cancer cells that survived the induction phase of treatment. For a portion of this phase, Rydapt is given with the chemotherapy drug cytarabine.

Rydapt with drugs to help prevent nausea and vomiting

During Rydapt treatment, your doctor will also prescribe certain medications to help reduce nausea and vomiting. Examples of these medications include:

• aprepitant (Emend, oral form)
• fosaprepitant (Emend, injectable form)
• ondansetron (Zofran, Zuplenz)
• granisetron (Sancuso, Sustol)
• palonosetron (Aloxi)
• dexamethasone
• olanzapine (Zyprexa)

Your doctor may prescribe more than one medication to help prevent or treat nausea and vomiting. During treatment, your doctor will monitor you for nausea and vomiting, and they’ll prescribe more than one medication if needed.

Rydapt and alcohol

There aren’t any known interactions between Rydapt and alcohol. However, many of the side effects caused by Rydapt can also be caused by alcohol.

For example, side effects that can occur with both Rydapt and alcohol include:

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

Drinking alcohol while taking Rydapt could increase your risk of these side effects. If you drink alcohol, talk with your doctor about how much alcohol is safe for you to consume during Rydapt treatment.

Rydapt interactions

Rydapt can interact with several other medications. It can also interact with certain supplements as well as certain foods.

Different interactions can cause different effects. For instance, some interactions can interfere with how well a drug works. Other interactions can increase side effects or make them more severe.

Rydapt and other medications

Below are lists of medications that can interact with Rydapt. These lists don’t contain all the drugs that may interact with Rydapt.

Before taking Rydapt, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions.

If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

Rydapt and certain HIV medications

Taking Rydapt with certain medications used to treat HIV can increase the level of Rydapt in your body. This can increase your risk of serious side effects from Rydapt.

Examples of HIV drugs that can increase your risk of serious side effects if taken with Rydapt include:

• boceprevir
• cobicistat (Tybost)
• nelfinavir (Viracept)
• ritonavir (Norvir)

Each of these drugs is available alone and in combination with other drugs that are used to treat HIV. If you take any combination medications for HIV, check to see if those medications contain any of the drugs listed above.

If you take any of drugs that are listed here with Rydapt, your doctor will monitor you for serious side effects. Your doctor may also prescribe different HIV treatment for you.

Rydapt and certain antibiotic and antifungal medications

Taking Rydapt with certain antibiotic or antifungal medications can increase the level of Rydapt in your body. This can increase your risk of serious side effects from Rydapt.

Examples of antibiotics that can increase your risk of serious side effects if taken with Rydapt include:

• clarithromycin (Biaxin XL)
• erythromycin (Ery-tab, EryPed)
• isoniazid

Examples of antifungals that can increase your risk of serious side effects if taken with Rydapt include:

• itraconazole (Onmel, Sporanox)
• ketoconazole (Extina, Ketozole, Xolegel)
• voriconazole (Vfend)

If you need to take any of these medications during Rydapt treatment, your doctor will monitor you for serious side effects. They may also prescribe a different antibiotic or antifungal drug for you.

Rydapt and certain seizure medications

Taking Rydapt with certain medications used to treat seizures can lower the level of Rydapt in your body. This can make Rydapt less effective in treating your condition.

Examples of seizure medications that can decrease Rydapt levels include:

• carbamazepine (Carbatrol, Epitol, Equetro, Tegretol)
• phenytoin (Dilantin, Phenytek)

It’s recommended that you don’t take Rydapt with any of these seizures medications. If you’re taking any of these drugs, your doctor may prescribe a different seizure treatment for you. Or they may recommend a treatment other than Rydapt for your condition.

Rydapt and herbs and supplements

There are certain herbs and supplements that may interact with Rydapt. Talk with your doctor or pharmacist before using any herbs or supplements while taking Rydapt.

Rydapt and St. John’s wort

Taking both Rydapt and the herbal supplement St. John’s wort can decrease the level of Rydapt in your body. This could make Rydapt less effective in treating your condition. You should avoid taking St. John’s wort while you’re using Rydapt.

Rydapt and foods

Certain foods may interact with Rydapt. If you have questions about foods you may need to avoid during Rydapt treatment, talk with your doctor.

Rydapt and grapefruit

Consuming grapefruit or grapefruit juice while you’re using Rydapt can increase the level of Rydapt in your body. This can increase the risk of serious side effects from the drug.

Talk with your doctor about whether it’s safe for you to consume small amounts of grapefruit or grapefruit juice while you’re using Rydapt.

Alternatives to Rydapt

Other drugs are available that can treat your condition. Some may be better suited for you than others. If you’re interested in finding an alternative to Rydapt, talk with your doctor. They can tell you about other medications that may work well for you.

Note: Some of the drugs listed below are used off-label to treat these specific conditions. With off-label use, a drug that’s approved to treat one condition is used to treat another condition.

Alternatives for acute myeloid leukemia

Examples of other drugs that may be used to treat acute myeloid leukemia (AML) include:

• cladribine
• fludarabine
• methotrexate (Otrexup, Rasuvo, Trexall)
• gemtuzumab (Mylotarg)
• enasidenib (Idhifa)
• sorafenib (Nexavar)

Alternatives for systemic mastocytosis

Examples of other drugs that may be used to treat systemic mastocytosis include:

• cladribine
• imatinib (Gleevec)
• interferon alfa-2b (Intron A)
• peginterferon alfa-2a (Pegasys)
• peginterferon alfa-2b (PegIntron)

Rydapt vs. Nexavar

You may wonder how Rydapt compares to other medications that are prescribed for similar uses. Here we look at how Rydapt and Nexavar are alike and different.

Ingredients

Rydapt contains the drug midostaurin, while Nexavar contains the drug sorafenib. Both of these drugs belong to a class of drugs called kinase inhibitors. (A class of drugs describes medications that work in the same way.)

Uses

Rydapt is approved to treat newly diagnosed acute myeloid leukemia (AML) in adults who have the FLT3 gene mutation (abnormal change). AML is a type of cancer that affects your blood and bone marrow. For this use, Rydapt is used in combination with chemotherapy drugs. (Chemotherapy describes traditional drugs used to treat cancer.)

In addition, Rydapt is approved to treat systemic mastocytosis in adults. With this condition, your body makes too many mast cells, which are a type of white blood cell. In some cases, systemic mastocytosis can lead to cancer. For this use, Rydapt can be given on its own.

Nexavar, on the other hand, is approved to treat certain types of cancer that affect your liver, kidneys, or thyroid. This drug isn’t approved to treat AML, but sometimes it’s used off-label in adults with the condition. (With off-label use, a drug that’s approved to treat one condition is used for another condition it isn’t approved to treat.) When used off-label to treat AML, Nexavar is typically given in combination with chemotherapy.

Drug forms and administration

Rydapt comes as capsules that are taken by mouth. It should be taken with food.

Nexavar comes as a tablets. But unlike Rydapt, it’s best to avoid taking Nexavar with food. Instead, Nexavar should be taken at least 1 hour before you eat or at least 2 hours after you’ve eaten.

Side effects and risks

Rydapt and Nexavar contain different drugs from the same drug class. Therefore, both medications can cause very similar side effects. Below are examples of these side effects.

Mild side effects

These lists contain examples of mild side effects that can occur with Rydapt, with Nexavar, or with both drugs (when taken individually).

• Can occur with Rydapt:
  • vomiting
  • headache
  • muscle or bone pain
  • upper respiratory infections, such as the common cold
• Can occur with Nexavar:
  • hair loss
  • weight loss
  • decreased appetite
• Can occur with both Rydapt and Nexavar:
  • diarrhea
  • nausea
  • infections
  • fatigue (lack of energy)
  • belly pain

Serious side effects

These lists contain examples of serious side effects that can occur with Rydapt, with Nexavar, or with both drugs (when taken individually).

• Can occur with Rydapt:
  • febrile neutropenia (a condition that causes both a fever and low levels of a certain type of white blood cell)
  • serious infections, such as urinary tract infection (UTI) and pneumonia
  • severe gastrointestinal problems, including bleeding in your digestive tract
  • pneumonitis (inflammation in your lungs) and lung disease
• Can occur with Nexavar:
  • high blood pressure
  • severe skin reactions, including hand-foot skin reaction and Stevens-Johnson Syndrome
  • hemorrhage (bleeding that doesn’t stop)
  • gastrointestinal perforation (a hole in the lining of your stomach or intestines)
  • heart problems, including abnormal heart rhythm and heart attack
  • liver damage and liver failure
• Can occur with both Rydapt and Nexavar:
  • severe allergic reaction

Effectiveness

Rydapt and Nexavar have different approved uses. But they’re both used to treat AML in adults who have the FLT3 gene mutation. However, keep in mind that while Rydapt is approved for this use, Nexavar is used off-label for the condition.

These drugs haven’t been directly compared to each other in clinical studies. But in separate studies, Rydapt and Nexavar were both effective in treating AML. In these studies, each of the drugs was used in combination with chemotherapy.

In addition, treatment guidelines list both drugs as options for people with AML who have the FLT3 gene mutation.

Costs

Rydapt and Nexavar are both brand-name drugs. There are currently no generic forms of either drug. Brand-name medications usually cost more than generics.

The actual price you’ll pay for either drug depends on your prescribed dosage, your insurance plan, your location, and the pharmacy you use.

Rydapt vs. cladribine

You may wonder how Rydapt compares to other medications that are prescribed for similar uses. Here we look at how Rydapt and cladribine are alike and different.

General

Rydapt contains the drug midostaurin, while cladribine is a generic drug. These medications belong to different drug classes, but they’re both used to treat certain types of blood-related cancer in adults. (A class of drugs describes medications that work in the same way.)

Specifically, an injectable form of cladribine is used to treat blood-related cancer. An oral form of cladribine is available as the brand-name drug Mavenclad. However, Mavenclad is only used to treat certain forms of multiple sclerosis (MS).

Uses

Rydapt is approved to treat newly diagnosed acute myeloid leukemia (AML) in adults who have the FLT3 gene mutation (abnormal change). AML is a type of cancer that affects your blood and bone marrow. For this use, Rydapt is given in combination with chemotherapy (traditional drugs used to treat cancer).

In addition, Rydapt is approved to treat systemic mastocytosis. With this condition, your body makes too many mast cells, which are a type of white blood cell. In some cases, systemic mastocytosis can lead to cancer. For this use, Rydapt can be used on its own.

Cladribine is approved to treat a different type of blood cancer called hairy cell leukemia. This drug isn’t approved to treat AML or systemic mastocytosis, but sometimes it’s used off-label for these conditions. (With off-label use, a drug that’s approved to treat one condition is used to treat another condition.)

Drug forms and administration

Rydapt comes as capsules that are taken by mouth. It should be taken with food.

Cladribine comes as a liquid solution that’s given by either:

• intravenous (IV) infusion that lasts several hours (with an IV infusion, the drug is injected into your vein over time)
• subcutaneous injection (an injection under your skin)

Side effects and risks

Rydapt and cladribine are different drugs, but they can cause similar side effects. Below are examples of these side effects.

More common side effects

These lists contain examples of more common side effects that can occur with Rydapt, with cladribine, or with both drugs (when taken individually).

• Can occur with Rydapt:
  • muscle or bone pain
  • upper respiratory infections, such as the common cold
• Can occur with cladribine:
  • decreased appetite
  • skin rash
  • sweating
  • injection site reaction (pain, redness, or swelling in the area near your injection)
• Can occur with both Rydapt and cladribine:
  • nausea
  • vomiting
  • diarrhea
  • belly pain
  • headache

Serious side effects

These lists contain examples of serious side effects that can occur with Rydapt, with cladribine, or with both drugs (when taken individually).

• Can occur with Rydapt:
  • severe gastrointestinal problems, including bleeding in your digestive tract
  • pneumonitis (inflammation in your lungs) and lung disease
  • serious infections, such as urinary tract infection (UTI)
• Can occur with cladribine:
  • reduced activity of your immune system, which leads to an increased risk of infections
  • anemia (low red blood cell level)
  • thrombocytopenia (low platelet level), which increases your risk of bleeding
  • nerve damage
  • kidney damage
• Can occur with both Rydapt and cladribine:
  • febrile neutropenia (a condition that causes both a fever and low levels of a certain type of white blood cell)
  • serious infections, such as pneumonia
  • severe allergic reaction

Effectiveness

Rydapt and cladribine have different approved uses, but they’re both used to treat AML and systemic mastocytosis.

These drugs haven’t been directly compared to each other in clinical studies. But in separate studies, both Rydapt and cladribine were effective in treating AML. And both Rydapt and cladribine were also effective in treating systemic mastocytosis.

In addition, current treatment guidelines for AML and systemic mastocytosis list both drugs as options for people with systemic mastocytosis.

Costs

Rydapt is a brand-name drug, while cladribine is a generic drug. Typically, brand-name medications cost more than generics.

However, Rydapt may be taken at home, while cladribine is given by a healthcare professional in a hospital or clinic. (This medication isn’t available for you to purchase at a pharmacy.) Medications given in a healthcare facility usually cost more than drugs that are taken at home.

The actual price you’ll pay for either drug depends on your prescribed dosage, your insurance plan, and whether you’re receiving your doses from a healthcare provider.

How to take Rydapt

You should take Rydapt according to your doctor’s or healthcare provider’s instructions.

When to take

Rydapt should be taken twice each day, about 12 hours apart. It doesn’t matter what time of day you take the drug, as long as your doses are about 12 hours apart.

Taking Rydapt with food

Rydapt should be taken with food. It might be convenient to take Rydapt with breakfast and dinner, since these meals are typically 12 hours apart.

Can Rydapt be crushed, split, or chewed?

No, Rydapt shouldn’t be crushed, split, or chewed. Crushing Rydapt capsules could change the safety and effectiveness of the drug.

Rydapt capsules should be swallowed whole. If you have trouble swallowing pills, talk with your doctor or pharmacist about other treatment options that are easier for you to take.

How Rydapt works

Rydapt is approved to treat newly diagnosed acute myeloid leukemia (AML). This condition is a type of cancer that affects your blood and bone marrow.

Rydapt is also approved to treat systemic mastocytosis. With this condition, your body overproduces certain blood cells called mast cells. In some cases, systemic mastocytosis can lead to cancer.

Below we describe how Rydapt treats both AML and systemic mastocytosis.

What Rydapt does for acute myeloid leukemia

AML is type of cancer that affects your blood and bone marrow. Normally, new blood cells are made in your bone marrow from cells called myeloid precursor cells. These precursor cells develop into white blood cells, red blood cells, and platelets. The precursor cells usually die when they’re old or they become damaged.

But with leukemia, you have abnormal myeloid precursor cells that don’t develop into normal, healthy blood cells. Instead, the abnormal precursor cells continue to grow and multiply without dying as they normally should. (Cells that multiply are making more cells.)

Some people with leukemia have a mutation (abnormal change) in their FLT3 gene. This mutation causes their body to make proteins called kinases. These proteins encourage the myeloid precursor cells to grow and multiply without dying. As a result, the abnormal (cancerous) precursor cells grow and spread in your body.

Rydapt belongs to a class of drugs called kinase inhibitors. Rydapt works by blocking (inhibiting) the activity of kinases. This helps to stop the cancer cells from growing and spreading.

What Rydapt does for systemic mastocytosis

With systemic mastocytosis, your body makes abnormally high levels of mast cells, which are a type of white blood cell. Mast cells are made in your bone marrow. Normally, they help your body fight off infections and heal wounds. And they usually die when they’re old or they become damaged.

In people with systemic mastocytosis, the mast cells grow and multiply without stopping. (Cells that multiply are making more cells.) The cells don’t die like usual. This leads to a large number of mast cells building up inside your body.

This buildup of mast cells can damage certain organs, including your bones, lymph nodes, liver, and intestines. The buildup can also lead to a rare form of cancer called mast cell leukemia.

Systemic mastocytosis is often caused by a mutation (abnormal change) in the KIT gene. This mutation causes mast cells to make a protein called KIT kinase. This protein encourages certain cells to grow and multiply without dying. As a result, mast cells grow abnormally and spread in your body.

Rydapt belongs to a class of drugs called kinase inhibitors. Rydapt works by blocking (inhibiting) the activity of kinases. This causes abnormal mast cells to die and helps stop their growth and spread.

How long does it take to work?

Rydapt begins working on both mutated cells and cancer cells soon after you start taking the drug. But it’s hard to know how long it takes to stop the cells from growing and spreading.

During clinical studies, Rydapt worked quickly in people with systemic mastocytosis. Within 2 weeks after starting the drug, half of the people taking Rydapt had either complete remission (absence of mutated cells) or partial remission (reduced activity of mutated mast cells).

It isn’t known for sure how quickly Rydapt will begin working inside your body. This will depend on your overall health and how your body responds to the drug. Your doctor can recommend ways to monitor your body’s response to Rydapt during treatment.

Rydapt and pregnancy

There aren’t enough studies in humans to know whether or not Rydapt is safe to take during pregnancy. However, animal studies have shown harm to fetuses when pregnant females were given the drug.

Keep in mind that animal studies don’t always predict what will happen in people. But because the drug’s risk to human pregnancies isn’t known for sure, women taking Rydapt should avoid pregnancy.

If you’re a female who could possibly become pregnant, your doctor will have you take a pregnancy test during the week before you start taking Rydapt. They’ll make sure you aren’t pregnant before you start using the drug.

Birth control is recommended for both men and women using Rydapt. For more information about this, see the section “Rydapt and birth control” below.

Rydapt’s pregnancy registry

The manufacturer of Rydapt has created a registry that gathers information about the safety of Rydapt use during pregnancy. Women are encouraged to contact the registry if they:

• are taking or have taken Rydapt during pregnancy
• either became pregnant or were already pregnant when exposed to a male sexual partner who’s taking Rydapt

The Rydapt pregnancy registry records health information about women and babies who may have been affected by Rydapt. This information helps doctors and other people using the drug to know more about the safety of Rydapt use during pregnancy.

More information can be found about the registry by calling 888-669-6682 or by visiting the manufacturer’s website.

In addition to joining this registry, tell your doctor right away if you become pregnant during Rydapt treatment.

Rydapt and birth control

Pregnancy should be avoided during Rydapt treatment.

Animal studies have shown harm to fetuses when the drug was given to pregnant females. Keep in mind that animal studies don’t always predict what will happen in humans. However, because of the possible risk to developing pregnancies, birth control use is recommended while you’re using Rydapt.

If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control options. Below we describe birth control recommendations for both males and females using Rydapt.

Birth control for females using Rydapt

If you’re a women taking Rydapt, and you’re able to become pregnant, you should use an effective form of birth control during treatment. You should continue using birth control for at least 4 months after your last dose of Rydapt.

Birth control for males using Rydapt

If you’re a man taking Rydapt, and you’re sexually active with a female who’s able to become pregnant, you should use effective birth control during treatment. And you should continue to use birth control for at least 4 months after your last dose of Rydapt. This is important to do even if your female partner is using birth control.

Rydapt and breastfeeding

It’s not known if Rydapt passes into human breast milk. However, in animal studies, the drug did pass into the breast milk of lactating animals.

Keep in mind that animal studies don’t always predict what will happen in humans. But because Rydapt can cause very serious side effects, breastfeeding isn’t recommended during treatment with the drug. And you should wait at least 4 months after your last dose of Rydapt to begin breastfeeding.

If you’re breastfeeding and considering taking Rydapt, talk with your doctor. They can recommend safe and healthy ways to feed your child.

Common questions about Rydapt

Here are answers to some frequently asked questions about Rydapt.

Is Rydapt a chemotherapy drug?

No, Rydapt isn’t considered a chemotherapy drug. Instead, it’s a targeted therapy that was made to work on specific traits of both mutated cells and cancer cells. (Depending on the condition you have, the affected cells have unique traits.)

Chemotherapy drugs work on cells that are quickly multiplying inside your body. (Cells that are multiplying are making more cells.) Both abnormal cells and some healthy cells multiply quickly. This means that chemotherapy can affect some of your healthy cells in addition to mutated cells and cancer cells.

Rydapt, on the other hand, is targeted to only work on certain parts of mutated cells and cancer cells. This means the drug doesn’t affect as many of your healthy cells as chemotherapy does. Because of this, Rydapt is also thought to have fewer side effects than chemotherapy drugs do.

Your doctor will order tests to determine the type of cancer you have. Then, they’ll choose the treatment that’s best for your condition. For some people, the recommended treatment might be a targeted treatment, such as Rydapt.

Will I have hair loss while taking Rydapt?

Yes, it’s possible that you might have hair loss with Rydapt treatment, but it may not be caused by Rydapt itself. Instead, it may be caused by a drug that’s used with Rydapt.

In clinical studies of Rydapt, hair loss wasn’t reported as a side effect. However, hair loss is commonly seen in people using certain chemotherapy drugs that are often used with Rydapt. (Chemotherapy describes traditional drugs used to treat cancer.)

For example, if you’re taking Rydapt to treat a certain type of leukemia, you’ll also be given a chemotherapy drug called daunorubicin. This drug may lead to hair loss in some people taking it.

If you’re concerned about hair loss during cancer treatment, talk with your doctor. They may have suggestions on how to help reduce this side effect.

What does “induction phase treatment” mean?

The induction phase is the first phase of acute myeloid leukemia (AML) treatment with Rydapt. This phase is also called remission induction.

The goal of the induction phase is to quickly kill as many cancer cells as possible. But during this phase, the treatment sometimes also kills many healthy blood cells. This can lead to serious side effects.

You’ll likely start the induction phase of AML treatment in the hospital. This way, your doctor will be able to oversee your medication dosing and monitor you for any side effects. Your dosage may need to be adjusted if you have severe side effects.

During the induction phase of AML treatment, you’ll receive Rydapt and two chemotherapy drugs: cytarabine and daunorubicin. The usual treatment cycle, which lasts 21 days, is as follows:

• Days 1 through 3. On these days, daunorubicin is given as an intravenous (IV) injection* once daily.
• Days 1 through 7. On these days, cytarabine is given as an IV infusion.**
• Days 8 through 21. On these days, Rydapt is taken by mouth twice each day.

* With an IV injection, the drug is injected into your vein all at once.

** With an IV infusion, the drug is injected into your vein over a period of time.

Depending on your other health conditions, you might receive chemotherapy drugs other than those listed above.

After the induction phase, your doctor may recommend an additional treatment phase. This second treatment phase is called the consolidation phase.

What does “consolidation phase treatment” mean?

The consolidation phase is the second phase of acute myeloid leukemia (AML) treatment with Rydapt. This phase is also called post-remission.

The consolidation phase is given after the induction phase. It’s used to help kill cancer cells that remain in your body after the first phase of treatment.

During the consolidation phase of AML treatment, you’ll receive high doses of a chemotherapy drug called cytarabine. The usual treatment cycle, which lasts 21 days, is as follows:

• Days 1, 3, and 5. On these days, high doses of cytarabine are given as IV infusions twice each day. These IV infusions each last for several hours.
• Days 8 through 21. On these days, Rydapt is taken by mouth twice each day.

Usually, the consolidation phase of AML treatment can be given in an outpatient clinic or in your home. But this will depend on whether you’re having any bothersome or severe side effects during treatment. Your doctor will help you decide if this is a good option, or if you should stay in the hospital instead.

Your doctor may recommend a total of three to four cycles of consolidation treatment. Their recommendation depends on how well your condition responds to treatment and how your body is handling the medications.

During the consolidation phase, your doctor may also recommend a stem cell transplant, if needed. With a stem cell transplant, you’ll receive stem cells that were collected either from someone else’s body or from your body at an earlier time.

This treatment option is usually used in people who are less than 60 years of age. It’s typically used if your condition isn’t improving enough with other therapies. Your doctor will recommend whether you’ll need to have a stem cell transfusion based on the risk of your leukemia coming back after treatment.

Should Rydapt be used in older adults with AML or mastocytosis?

Yes, Rydapt can be a treatment option for older adults. In fact, Rydapt is approved for use in adults, including those ages 65 years and older, who have newly diagnosed acute myeloid leukemia (AML). This condition is a type of cancer that affects your blood and bone marrow.

Rydapt is also approved for use in adults, including those ages 65 years and older, with systemic mastocytosis. With this condition, your body makes too many mast cells, which are a type of white blood cell. In some cases, systemic mastocytosis can lead to cancer.

However, depending on your age and any other health conditions you may have, your doctor may adjust your treatment by having you:

• take a lower dosage of Rydapt than the dosage that’s typically used
• take a lower dosage of the chemotherapy drugs that are typically used with Rydapt
• take Rydapt with chemotherapy drugs other than those typically used

Your doctor will prescribe a treatment combination that’s safe for you to use based on your age and other health conditions you may have.

Rydapt precautions

Before taking Rydapt, talk with your doctor about your health history. Rydapt may not be right for you if you have certain medical conditions. This includes:

• History of allergic reaction to Rydapt. You shouldn’t take Rydapt if you’ve had an allergic reaction to the medication or to any of its ingredients in the past. If you’re allergic to Rydapt, talk with your doctor about other treatment options. If you’re not sure about your medication allergies, talk with your doctor.

Note: For more information about the potential negative effects of Rydapt, see the “Rydapt side effects” section above.

Rydapt overdose

Don’t use more Rydapt than your doctor recommends.

There weren’t any overdoses reported during clinical trials of Rydapt. Because of this, it’s not known what side effects an overdose may cause. However, your risk of serious side effects is increased with an overdose.

What to do in case you take too much Rydapt

If you think you’ve taken too much of this drug, call your doctor. You can also call the American Association of Poison Control Centers at 800-222-1222 or use their online tool. But if your symptoms are severe, call 911 or go to the nearest emergency room right away.

Rydapt expiration, storage, and disposal

When you get Rydapt from the pharmacy, the pharmacist will add an expiration date to the label on the bottle. This date is typically 1 year from the date they dispensed the medication.

The expiration date helps guarantee the effectiveness of the medication during this time. The current stance of the Food and Drug Administration (FDA) is to avoid using expired medications. If you have unused medication that has gone past the expiration date, talk to your pharmacist about whether you might still be able to use it.

Storage

How long a medication remains good can depend on many factors, including how and where you store the medication.

Rydapt capsules should be stored at room temperature (68°F to 77°F/20°C to 25°C). The drug should be kept in a tightly sealed container, protected from moisture. Avoid storing this medication in areas where it could get damp or wet, such as in bathrooms.

Disposal

If you no longer need to take Rydapt and have leftover medication, it’s important to dispose of it safely. This helps prevent others, including children and pets, from taking the drug by accident. It also helps keep the drug from harming the environment.

The FDA website provides several useful tips on medication disposal. You can also ask your pharmacist for information on how to dispose of your medication.