Rydapt (midostaurin)
An approved treatment for Systemic Mastocytosis and Mastocytosis.
The same compound appears under different names depending on the context. Here is how to identify Midostaurin wherever you encounter it, plus the key facts at a glance.
- Generic name
- Midostaurin
- Brand name
- Rydapt
- Development code
- PKC412
- Drug class
- Kinase inhibitor (multi-kinase)
- Manufacturer
- Novartis
- How it's taken
- For advanced systemic mastocytosis, 100 mg by mouth twice a day, about 12 hours apart, with food.
A capsule taken twice a day with food for adults with advanced systemic mastocytosis: aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated blood cancer (SM-AHN) or mast cell leukemia (MCL). The FDA approved it on April 28, 2017, at the same time as an approval for newly diagnosed FLT3-positive acute myeloid leukemia alongside chemotherapy. It is not approved for indolent systemic mastocytosis.
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Where Midostaurin fits
One of 2 approved targeted treatments for advanced systemic mastocytosis, with avapritinib (Ayvakit). Nausea and vomiting are frequent, so anti-nausea medicine is part of taking it.
How Midostaurin works
Midostaurin blocks several signaling enzymes at once. In lab studies, it and its active breakdown products blocked KIT, both the normal form and the D816V form that drives most systemic mastocytosis, as well as FLT3, PDGF receptors and protein kinase C[1].
Mechanism: Oral multi-kinase inhibitor that blocks KIT, including the D816V form, and FLT3, among other kinases
Side effects and safety
- Lung inflammation. Interstitial lung disease and pneumonitis have occurred, some fatal. Report new cough, shortness of breath or chest discomfort; Rydapt is stopped if lung inflammation without infection develops.
- Harm to an unborn baby. A pregnancy test is done within 7 days before starting. Use effective birth control during treatment and for 4 months after the last dose, for both women and men with partners who could become pregnant.
- Allergic reactions. Do not take Rydapt if you are allergic to midostaurin. Reactions have included anaphylactic shock, flushing, chest pain and swelling of the airways or tongue.
- Checkups at least weekly for the first 4 weeks, every other week for the next 8 weeks, then monthly
- Blood counts, since the dose is paused for low white cells, platelets or hemoglobin
- Heart tracings (ECG) when taken with other medicines that affect the QT interval
- Pregnancy test within 7 days before starting
Rydapt has no boxed warning. People allergic to midostaurin must not take it; reactions have included anaphylactic shock and swelling of the airways. The label warns about lung inflammation (interstitial lung disease or pneumonitis), which has been fatal in some cases, and harm to an unborn baby. In systemic mastocytosis, side effects were common: nausea (82%), vomiting (68%), diarrhea (54%), swelling (40%), muscle and bone pain (35%), stomach pain (34%), tiredness (34%) and upper respiratory infections (30%). A QT change on the heart tracing occurred in 11%[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Midostaurin
For advanced systemic mastocytosis, 100 mg by mouth twice a day, about 12 hours apart, with food. Anti-nausea medicine is given beforehand to lower the risk of nausea and vomiting. Capsules are swallowed whole, not opened or crushed, and a missed or vomited dose is skipped. Strong CYP3A4 inhibitors raise midostaurin levels, so alternatives are considered, and strong CYP3A4 inducers should be avoided[1].
Availability and cost
Only available as the brand-name product.
Help paying for Rydapt
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Novartis Oncology Universal Co-Pay Program: for patients with private insurance, with a combined annual limit up to $15,000. Not valid with Medicare, Medicaid, TRICARE, VA, DoD or other federal or state programs.
For: private insurance · source
Good to know: Novartis lists Rydapt capsules among its products with co-pay programs. The program's per-fill amount for Rydapt is not shown on Novartis's pages, and a free-drug option is not listed here until confirmed on Novartis's own pages.
- From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)Acute Myeloid Leukemia Co-Pay Fund (covered diagnoses include Mastocytosis) fundOpen
Pays for: Insurance premiums and treatment-related copays, deductibles and coinsurance, up to $4,000 per year. Requires health insurance (any kind).
Access and eligibility
Approved for adults with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm or mast cell leukemia. Not approved for indolent or smoldering systemic mastocytosis.
Source: Novartis Oncology co-pay program
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
The main systemic mastocytosis trial (NCT00782067), with no comparison group, enrolled 116 adults with advanced systemic mastocytosis who had received up to 2 earlier treatments; 89 could be assessed for response, and 82% of them carried KIT D816V. By 6 cycles, 21% had a complete or incomplete remission by modified Valent criteria: 38% with ASM, 16% with SM-AHN and 25% with MCL. Major or partial responses occurred in 46 of 73 patients with KIT D816V and 7 of 16 without it or with unknown status[1][3].
Development history
Novartis developed midostaurin under the code PKC412. The FDA approved Rydapt on April 28, 2017 for advanced systemic mastocytosis and for newly diagnosed FLT3-positive acute myeloid leukemia with chemotherapy[2].
Explore Systemic Mastocytosis and Mastocytosis trials
Other Systemic Mastocytosis & Mastocytosis treatments
For advanced systemic mastocytosis, Rydapt (approved 2017) and Ayvakit (approved for advanced disease in 2021) are the FDA-approved targeted treatments. Ayvakit is also approved for indolent disease. Cogent's bezuclastinib has an FDA decision due June 29, 2027 for advanced systemic mastocytosis.
Common questions about Midostaurin
▸What is Midostaurin (Rydapt)?
A capsule taken twice a day with food for adults with advanced systemic mastocytosis: aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated blood cancer (SM-AHN) or mast cell leukemia (MCL). The FDA approved it on April 28, 2017, at the same time as an approval for newly diagnosed FLT3-positive acute myeloid leukemia alongside chemotherapy. It is not approved for indolent systemic mastocytosis.
▸How does Midostaurin work?
Midostaurin blocks several signaling enzymes at once. In lab studies, it and its active breakdown products blocked KIT, both the normal form and the D816V form that drives most systemic mastocytosis, as well as FLT3, PDGF receptors and protein kinase C[1].
▸What are the side effects of Midostaurin?
Rydapt has no boxed warning. People allergic to midostaurin must not take it; reactions have included anaphylactic shock and swelling of the airways. The label warns about lung inflammation (interstitial lung disease or pneumonitis), which has been fatal in some cases, and harm to an unborn baby. In systemic mastocytosis, side effects were common: nausea (82%), vomiting (68%), diarrhea (54%), swelling (40%), muscle and bone pain (35%), stomach pain (34%), tiredness (34%) and upper respiratory infections (30%). A QT change on the heart tracing occurred in 11%[1].
▸How is Midostaurin taken?
For advanced systemic mastocytosis, 100 mg by mouth twice a day, about 12 hours apart, with food. Anti-nausea medicine is given beforehand to lower the risk of nausea and vomiting. Capsules are swallowed whole, not opened or crushed, and a missed or vomited dose is skipped. Strong CYP3A4 inhibitors raise midostaurin levels, so alternatives are considered, and strong CYP3A4 inducers should be avoided[1].
▸Is Midostaurin FDA approved?
Yes, Midostaurin (Rydapt) is FDA approved (2017) for the treatment of Systemic Mastocytosis and Mastocytosis.
▸Is Rydapt used for indolent systemic mastocytosis?
No. Rydapt is approved only for advanced forms: aggressive systemic mastocytosis, systemic mastocytosis with an associated blood cancer and mast cell leukemia. Ayvakit (avapritinib) is the approved targeted pill for indolent systemic mastocytosis.
▸How do you manage nausea on Rydapt?
The label calls for anti-nausea medicine before treatment and taking the capsules with food. In the systemic mastocytosis trials, 82% had nausea and 68% vomiting; nausea started a median of 9 days into treatment.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2026-06. RYDAPT (midostaurin) capsules: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=11fa3fc9-6776-49a6-b1c1-653f627c3e58
- U.S. Food and Drug Administration. Rydapt approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=207997
- ClinicalTrials.gov. Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia. https://clinicaltrials.gov/study/NCT00782067
- Novartis. Available co-pay programs. https://www.novartis.com/us-en/sites/novartis_us/files/patient-support-options.pdf