Midostaurin
The first drug to improve survival in FLT3-mutated AML, added to standard chemotherapy: median survival went from about two years to more than six.
RATIFY (n=717, FLT3-ITD or TKD, age 18-59): midostaurin with 7+3 and as maintenance improved OS (median 74.7 vs 25.6 months, HR 0.78). Approved 2017 for newly diagnosed FLT3-mutated AML and for systemic mastocytosis. Now being displaced in FLT3-ITD by quizartinib (QuANTUM-First) and challenged by gilteritinib in frontline trials; still the only option labelled for FLT3-TKD.
1.Midostaurin binds the active conformation of FLT3
- Route
- Oral
- Schedule
- 50 mg twice daily on days 8-21 of each induction and consolidation cycle, then 50 mg twice daily continuously for up to 12 cycles of maintenance
- Monitoring
- QT interval, pulmonary symptoms, counts
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
- Medicare
- Part D (self-administered)
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Oral, taken with induction chemotherapy; inpatient doses are bundled into the Part A stay, outpatient supply is Part D.
- Commercial insurance
- covered with prior authorisation
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. FLT3 mutation by an approved test.
- Assistance programmes
- Novartis Patient Support (Patient Assistance NOW Oncology)
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
- Leukemia & Lymphoma Society financial support
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Appraised for
- Untreated FLT3 mutation-positive AML with standard chemotherapy
- NHS England
- Routinely funded for the appraised indication (or via managed access)
Sources: NICE TA523 · SMC advice: midostaurin. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
- 28 Apr 2017ApprovalUS
First FLT3 inhibitor approved
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2017 | Newly diagnosed FLT3-mutated AML with 7+3 and consolidation; advanced systemic mastocytosis |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Febrile neutropenia Both arms of RATIFY; not drug-specific | — | 84% |
| Nausea | 83% | — |
| Rash/desquamation | — | 14% |
| QT prolongation Monitor; avoid strong CYP3A4 inhibitors where possible | — | — |
Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
QuANTUM-First gave FLT3-ITD AML patients a second front-line targeted option and showed that continuing a FLT3 inhibitor as long-term maintenance, including after transplant, pays off. Quizartinib was approved for this indication in 2023. Head-to-head data against midostaurin are lacking, and the design leaves open how much of the benefit came from maintenance.
ADMIRAL showed that a targeted oral drug can beat chemotherapy outright in relapsed AML, and made gilteritinib the standard bridge to transplant for FLT3-mutated relapse. Its success also underpinned FLT3 inhibitor use in first-line combinations. Resistance through FLT3-independent clones and RAS pathway mutations limits durability without transplant.
Latest papers
topQuery for this drug: (TITLE:"Midostaurin" OR ABSTRACT:"Midostaurin" OR TITLE:"Rydapt" OR ABSTRACT:"Rydapt") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Midostaurin, not a curated reading list.