RYDAPT (midostaurin)
RYDAPT is indicated for the treatment of Acute Myeloid Leukemia; Aggressive Systemic Mastocytosis; Systemic Mastocytosis with Associated Hematological Neoplasm; Mast Cell Leukemia.
How RYDAPT Works
Midostaurin and its major active metabolites inhibit multiple receptor tyrosine kinases, including wild-type FLT3, FLT3 mutation kinases (ITD and TKD), KIT (wild-type and D816V mutant), PDGFRα/β, and members of the serine/threonine kinase PKC (protein kinase C) family. By inhibiting these signaling pathways, midostaurin inhibits cell proliferation and induces apoptosis in leukemic cells and mast cells. In mast cells, it specifically inhibits KIT signaling and the release of histamine.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2017-04-28
- Patent Cliff
- 2030
- Routes
- ORAL
- Dosage Forms
- CAPSULE
RYDAPT Approval History
What RYDAPT Treats
4 indicationsRYDAPT is approved for 4 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute Myeloid Leukemia
- Aggressive Systemic Mastocytosis
- Systemic Mastocytosis with Associated Hematological Neoplasm
- Mast Cell Leukemia
RYDAPT Target & Pathway
ProTarget
RYDAPT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in RYDAPT's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications RYDAPT treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to RYDAPT
3 of 19FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
29 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03591510 | CPKC412A2218 2017-004830-28 | Ph 2 | active not recruiting | A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML |
| NCT03258931 | ARO-021 | Ph 3 | completed | Study of Crenolanib vs Midostaurin Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed FLT3 Mutated AML |
| NCT06313437 | 24-021 | Ph 1 | recruiting | Revumenib in Combination With 7+3 + Midostaurin in AML |
| NCT02115295 | 2012-0648 NCI-2014-01103, 2012-0648 | Ph 2 | recruiting | Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia |
| NCT03900949 | OSU-21190 NCI-2019-01726 | Ph 1 | completed | Gentuzumab Ozogamicin and Midostaurin Combination With Standard Cytarabine and Danunorubi Midostaurin as a Novel Approach to Treating Patients With Newly Diagnosed FLT-3 Mutated Acute Myeloid Leukemia |
| NCT03836209 results posted | PrE0905 | Ph 2 | active not recruiting | Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia |
| NCT03280030 results posted | CPKC412A2220 | Ph 2 | completed | A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML |
| NCT03686345 AML FLT3 | REL-AML 001/2017 2017-002094-18 | Ph 2 | terminated | Midostaurin Associated With Standard Chemotherapy in Patients With Core-binding Factor Leukemia |
| NCT03092674 results posted | NCI-2017-00436 NCI-2017-00436, S1612 | Ph 2, Ph 3 | completed | Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome |
| NCT04027309 HOVON 156 AML | HO156 2018-000624-33, AMLSG 28-18 | Ph 3 | active not recruiting | A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy |
| NCT04982354 | 2019-KOE-003 | Ph 1, Ph 2 | withdrawn | Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia |
| NCT04097470 HO155 | HOVON 155 AML 2018-000047-31, 2018-674 | Ph 2 | active not recruiting | Tolerability and Efficacy of Midostaurin to 10-day Decitabine in Unfit Adult AML and High Risk MDS Patients |
| NCT03379727 results posted | CPKC412A2408 2016-004440-12 | Ph 3 | completed | Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Combination With Standard Chemotherapy During Induction and Consolidation Followed by 12 Months of Maintenance Monotherapy in Patients With Newly-diagnosed FMS-like Tyrosine 3 (FLT3) Kinase Receptor-mutated Acute Myeloid Leukemia. |
| NCT04075747 V-FAST | JZP025-101 | Ph 1 | completed | A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia |
| NCT03512197 results posted | CPKC412E2301 2017-003540-21 | Ph 3 | completed | A Global Study of the Efficacy and Safety of Midostaurin + Chemotherapy in Newly Diagnosed Patients With FLT3 Mutation Negative (FLT3-MN) Acute Myeloid Leukemia (AML) |
| NCT04496999 HDMM | HDMM | Ph 1 | terminated | HDM201 and Midostaurin (HDMM) in Relapsed/Refractory AML With FLT3mut and TP53wt. |
| NCT01093573 results posted | CASE1908 NCI-2009-01285 | Ph 1, Ph 2 | completed | Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia |
| NCT03951961 | MAURITIUS 2019-000136-26 | Ph 2 | terminated | Midostaurin in MRD (Minimal Residual Disease) Positive Acute Myeloid Leukemia After Allogeneic Stem Cell Transplantation |
| NCT01429337 | CPKC412A2116 2010-020694-16 | Ph 1 | completed | PK and Safety of Midostaurin in Subjects With Impaired Hepatic Function and Subjects With Normal Hepatic Function |
| NCT01883362 RADIUS results posted | CPKC412AUS23 | Ph 2 | completed | Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML |
| NCT01477606 | AMLSG 16-10 2011-003168-63 | Ph 2 | completed | Protocol in Acute Myeloid Leukemia With FLT3-ITD |
| NCT03760445 | CHDM201A2101 2018-003107-19 | Ph 1, Ph 2 | withdrawn | HDM201 Added to CT in R/R or Newly Diagnosed AML |
| NCT02634827 results posted | MC1483 NCI-2015-02107, MC1483 | Ph 2 | terminated | Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation |
| NCT01282502 | 10-457 | Ph 1 | completed | Midostaurin (PKC412) for Locally Advanced Rectal Cancer |
| NCT03207334 | UF-HEM-004 CPKC412AUS61T, IRB201701934 | Ph 2 | withdrawn | iCare4: Genomic Signatures With Midostaurin in Acute Myeloid Leukemia (UF-HEM-004) |
| NCT01846624 results posted | IRB-25737 HEMAML0022 | Ph 2 | terminated | Decitabine and Midostaurin in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT01174888 | OSU-09111 NCI-2010-01335 | Ph 1 | completed | Phase I Combination of Midostaurin, Bortezomib, and Chemo in Relapsed/Refractory Acute Myeloid Leukemia |
| NCT00866281 results posted | CPKC412A2114 2008-006931-11 | Ph 1, Ph 2 | terminated | A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia |
| NCT01161550 CLAG ATRA AML | 10-1181 / 201108160 | Ph 1 | completed | Cladribine Based Induction Therapy With All-Trans Retinoic Acid and Midostaurin in Relapsed/Refractory AML |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
RYDAPT FDA Label Details
Indications & Usage
FDA Label (PDF)RYDAPT is indicated for the treatment of Acute Myeloid Leukemia; Aggressive Systemic Mastocytosis; Systemic Mastocytosis with Associated Hematological Neoplasm; Mast Cell Leukemia.
Pro Intelligence Preview
Deep insights for RYDAPT
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2030
- • 2 active patents
Trial Analysis
- • 30 total trials
- • Stage: Stable
Competitive Landscape
- • 19 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment