Rydapt (midostaurin)
An approved treatment for Acute Myeloid Leukemia.
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, FLT3)
- Manufacturer
- Novartis
- How it's taken
- Oral capsule twice daily on days 8 to 21 of each chemotherapy cycle
A capsule taken twice a day on days 8 to 21 of each chemotherapy cycle, added to standard 7+3 induction and high-dose cytarabine consolidation for adults with newly diagnosed AML that carries a FLT3 mutation. Approved April 28, 2017 as the first targeted drug for AML. Rydapt is also approved for advanced systemic mastocytosis, covered on a separate page.
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Where Midostaurin fits
One of 2 FLT3 inhibitors approved with induction chemotherapy. Rydapt covers FLT3-ITD and FLT3-TKD mutations; Vanflyta (quizartinib) covers FLT3-ITD only but continues as maintenance after consolidation. They have not been compared head to head.
How Midostaurin works
About 3 in 10 adults with AML have a change in the FLT3 gene, most often an internal tandem duplication (ITD), that keeps a growth signal switched on in the leukemia cells. Midostaurin and its active breakdown products block FLT3 in both its ITD and TKD forms, as well as KIT, PDGF receptors, VEGFR2 and protein kinase C[1]. Because it is a broad kinase blocker rather than a FLT3-specific one, it is given only on days 8 to 21 of each cycle, after the chemotherapy days.
Mechanism: Oral multi-kinase inhibitor that blocks FLT3 (ITD and TKD forms) along with KIT, PDGFR and other kinases
Side effects and safety
- Pulmonary toxicity. Interstitial lung disease and pneumonitis, some fatal; new cough or breathlessness is reported at once and Rydapt is stopped if lung inflammation without infection is found.
- Embryo-fetal toxicity. Pregnancy test within 7 days before starting; effective contraception during treatment and for 4 months after, for women and for men with partners who could become pregnant.
- Allergic reactions. Do not take Rydapt if allergic to midostaurin; anaphylaxis has occurred.
- Blood counts and chemistry as part of the chemotherapy schedule
- ECG when Rydapt is combined with other medicines that prolong the QT interval
- Pregnancy test within 7 days before the first dose
- Symptoms of lung inflammation
Rydapt has no boxed warning. Its warnings are harm to an unborn baby (a pregnancy test within 7 days of starting) and pulmonary toxicity, meaning interstitial lung disease or pneumonitis that has been fatal in some cases[1].
In the RATIFY trial the side effects that were at least 2 percentage points more common with Rydapt than placebo were nausea (83% vs 70%), mucositis (66% vs 62%), vomiting (61% vs 53%), headache (46% vs 38%), petechiae (36% vs 27%), musculoskeletal pain, nosebleeds, device-related infections (24% vs 17%), high blood sugar and a prolonged QT interval on the ECG (20% vs 17%)[1]. Febrile neutropenia occurred in 83% of both arms, which is a feature of the chemotherapy[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 AML, 50 mg by mouth twice a day with food on days 8 to 21 of each cycle of induction with cytarabine and daunorubicin (up to 2 cycles) and on days 8 to 21 of each cycle of consolidation with high-dose cytarabine (up to 4 cycles)[1]. In the RATIFY trial, patients who did not go to transplant continued Rydapt alone for up to 12 more 28-day cycles, which the label describes but does not list as an indication; single-agent Rydapt is not approved as induction[1].
Anti-nausea medicine is given before doses, and strong CYP3A4 inhibitors such as azole antifungals raise midostaurin levels[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: Copied from the existing midostaurin (mastocytosis) record checked October 12, 2026 so the AML page shows the same program. Novartis lists Rydapt among its co-pay products; the per-fill amount is not shown and a free-drug option is not listed until confirmed on Novartis's own pages.
Access and eligibility
Requires a FLT3 mutation (ITD or TKD) found by an FDA-approved test, and is given only with standard cytarabine and daunorubicin induction and cytarabine consolidation in newly diagnosed disease. It is not approved alone, and it is not approved for relapsed AML, where Xospata is the FLT3 option.
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.
How the targeted AML pills compare, by mutation
AML has 19 FDA-approved medicines, counting the 3 older chemotherapy drugs. This chart covers the 8 that target a specific gene change, because the result of your mutation test decides which of them could apply to you. Chemotherapy (7+3, Vyxeos), Venclexta, Mylotarg, Onureg, Daurismo and the APL drugs are described on the drug pages instead. Several of these drugs are only approved after relapse, and all of the FLT3, IDH and menin drugs can cause differentiation syndrome, so your leukemia doctor will weigh the mutation, your point in treatment and your heart and liver tests.
Acute Myeloid Leukemia has 8 FDA-approved treatments: Rydapt (midostaurin, approved 2017); Vanflyta (quizartinib, approved 2023); Xospata (gilteritinib, approved 2018); Tibsovo (ivosidenib, approved 2018); Rezlidhia (olutasidenib, approved 2022); Idhifa (enasidenib, approved 2017); Revuforj (revumenib, approved 2024); Komzifti (ziftomenib, approved 2025). Rydapt is typically used as newly diagnosed, with intensive chemotherapy.
| Drug | Mutation it targets | When it is approved | How it's taken | How often | Given with | FDA approved |
|---|---|---|---|---|---|---|
Rydapt midostaurin You are here | FLT3 (ITD or TKD) | Newly diagnosed, with intensive chemotherapy | Oral capsule | Twice daily on days 8 to 21 of each chemotherapy cycle | 7+3 induction and high-dose cytarabine consolidation (hospital-based chemotherapy) | 2017 |
Vanflyta quizartinib | FLT3-ITD only | Newly diagnosed, with intensive chemotherapy, then maintenance | Oral tablet | Once daily, with chemotherapy and then up to 36 months of maintenance | 7+3 induction and cytarabine consolidation, then alone as maintenance (not after transplant) | 2023 |
Xospata gilteritinib | FLT3 (ITD or TKD) | Relapsed or refractory | Oral tablets | Once daily | Taken alone | 2018 |
Tibsovo ivosidenib | IDH1 | Newly diagnosed (older or unfit) and relapsed or refractory | Oral tablets | Once daily | With azacitidine or alone (newly diagnosed, 75+ or unfit); alone at relapse | 2018 |
Rezlidhia olutasidenib | IDH1 | Relapsed or refractory | Oral capsule | Twice daily on an empty stomach | Taken alone | 2022 |
Idhifa enasidenib | IDH2 | Relapsed or refractory | Oral tablet | Once daily | Taken alone | 2017 |
Revuforj revumenib | KMT2A rearrangement (any acute leukemia) or NPM1 mutation (AML) | Relapsed or refractory (NPM1: after other options) | Oral tablets | Twice daily | Taken alone; adults and children 1 year and older | 2024 |
Komzifti ziftomenib | NPM1 mutation | Relapsed or refractory, after other options | Oral capsules | Once daily on an empty stomach | Taken alone; adults only | 2025 |
This chart is for education only and is not medical advice. Approval dates, indications and warnings come from each drug's FDA prescribing information and Drugs@FDA. None of these drugs has been compared head to head with another in the same column, and trial results are not comparable across different studies. A leukemia specialist who has your mutation report will know which, if any, applies to you and whether a clinical trial would fit better.
Clinical trial results
RATIFY (Study 1, NCT00651261) randomized 717 adults aged 18 to 60 with newly diagnosed FLT3-mutated AML to midostaurin or placebo added to 7+3 induction and high-dose cytarabine consolidation, followed by up to 12 cycles of the study drug alone. After a minimum follow-up of about 3.5 years, overall survival was better with midostaurin (hazard ratio 0.77, 95% CI 0.63 to 0.95, p=0.016); the survival curves flattened before reaching the median, so the label does not give a median.
Median event-free survival was 8.2 months against 3.0 months[1][2]. The trial enrolled people up to age 60; the label does not set an upper age limit.
Development history
Midostaurin was developed by Novartis under the code PKC412. The FDA approved it on April 28, 2017 for FLT3-mutated AML together with the LeukoStrat CDx FLT3 Mutation Assay as the companion test, and on the same day for advanced systemic mastocytosis[3]. The label was last revised in June 2026 with no change to the AML indication[1].
Explore Acute Myeloid Leukemia trials
Other Acute Myeloid Leukemia treatments
FLT3 testing at diagnosis decides whether Rydapt or Vanflyta is added to 7+3. At relapse, Xospata (gilteritinib) is the approved FLT3 inhibitor, and trials are testing FLT3 inhibitors combined with venetoclax and with menin inhibitors.
Common questions about Midostaurin
▸What is Midostaurin (Rydapt)?
A capsule taken twice a day on days 8 to 21 of each chemotherapy cycle, added to standard 7+3 induction and high-dose cytarabine consolidation for adults with newly diagnosed AML that carries a FLT3 mutation. Approved April 28, 2017 as the first targeted drug for AML. Rydapt is also approved for advanced systemic mastocytosis, covered on a separate page.
▸How does Midostaurin work?
About 3 in 10 adults with AML have a change in the FLT3 gene, most often an internal tandem duplication (ITD), that keeps a growth signal switched on in the leukemia cells. Midostaurin and its active breakdown products block FLT3 in both its ITD and TKD forms, as well as KIT, PDGF receptors, VEGFR2 and protein kinase C[1]. Because it is a broad kinase blocker rather than a FLT3-specific one, it is given only on days 8 to 21 of each cycle, after the chemotherapy days.
▸What are the side effects of Midostaurin?
Rydapt has no boxed warning. Its warnings are harm to an unborn baby (a pregnancy test within 7 days of starting) and pulmonary toxicity, meaning interstitial lung disease or pneumonitis that has been fatal in some cases[1]. In the RATIFY trial the side effects that were at least 2 percentage points more common with Rydapt than placebo were nausea (83% vs 70%), mucositis (66% vs 62%), vomiting (61% vs 53%), headache (46% vs 38%), petechiae (36% vs 27%), musculoskeletal pain, nosebleeds, device-related infections (24% vs 17%), high blood sugar and a prolonged QT interval on the ECG (20% vs 17%)[1]. Febrile neutropenia occurred in 83% of both arms, which is a feature of the chemotherapy[1].
▸How is Midostaurin taken?
For AML, 50 mg by mouth twice a day with food on days 8 to 21 of each cycle of induction with cytarabine and daunorubicin (up to 2 cycles) and on days 8 to 21 of each cycle of consolidation with high-dose cytarabine (up to 4 cycles)[1]. In the RATIFY trial, patients who did not go to transplant continued Rydapt alone for up to 12 more 28-day cycles, which the label describes but does not list as an indication; single-agent Rydapt is not approved as induction[1]. Anti-nausea medicine is given before doses, and strong CYP3A4 inhibitors such as azole antifungals raise midostaurin levels[1].
▸Is Midostaurin FDA approved?
Yes, Midostaurin (Rydapt) is FDA approved (2017) for the treatment of Acute Myeloid Leukemia.
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.
- Novartis via openFDA · 2026-06-08. Rydapt (midostaurin) capsules prescribing information. https://api.fda.gov/drug/label.json?search=set_id:%2211fa3fc9-6776-49a6-b1c1-653f627c3e58%22
- New England Journal of Medicine · 2017-08-03. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation. https://pubmed.ncbi.nlm.nih.gov/28644114/
- FDA. Rydapt approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=207997