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Drug

Midostaurin

Approved · EMA
Emerging researchLate-stage development

Also known as 4'-N-benzoylstaurosporine, Rydapt.

RxNorm1919083ChEMBLCHEMBL608533UNIIID912S5VON
2
Research papers
6
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase II, Open-label, Single Arm Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy and as a Single Agent Post-consolidation Therapy in Children With Untreated FLT3-mutated AML

Clinical trial2026-07-09Results expected Q2 2029 · ClinicalTrials.gov

Randomized Trial of Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia (AML)

Clinical trial2026-06-23Results posted · ClinicalTrials.gov

Phase II Study of Cladribine Plus Idarubicin Plus Cytarabine (ARAC) in Patients With AML, HR MDS, or Myeloid Blast Phase of CML

Clinical trial2026-05-20Results expected Q2 2030 · ClinicalTrials.gov

Phase III Randomized Study of Crenolanib Versus Midostaurin Administered Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed Subjects With FLT3 Mutated Acute Myeloid Leukemia

Clinical trial2026-04-06Completed · ClinicalTrials.gov

A Phase I Trial of Revumenib in Combination With 7+3 (7 Days of Cytarabine and 3 Days of Daunorubicin) + Midostaurin Induction Chemotherapy for the Frontline Treatment of NPM1 and FLT3 Mutated AML

Clinical trial2026-03-25Results expected Q1 2027 · ClinicalTrials.gov

A Phase I Study to Evaluate the Safety and Tolerability of Gemtuzumab Ozogamicin and Midostaurin When Used in Combination With Standard Cytarabine and Daunorubicin Induction for Newly Diagnosed FLT3-mutated Acute Myeloid Leukemia

Clinical trial2025-10-23Completed · ClinicalTrials.gov

A Phase 3, Multicenter, Open-label, Randomized, 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 (AML) or Myelodysplastic Syndromes With Excess Blasts-2 (MDS-EB2) With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML / AMLSG 28-18)

Clinical trial2025-10-01Primary completion · ClinicalTrials.gov

A Phase II, Randomized, Double-blind, Multi-center, Placebo-controlled Study to Evaluate the Efficacy and Safety of Twice Daily Oral Midostaurin in Combination With Daunorubicin/Cytarabine Induction, High-dose Cytarabine Consolidation, and Midostaurin Single Agent Continuation Therapy in Newly Diagnosed Patients With FLT3-mutated Acute Myeloid Leukemia (AML).

Clinical trial2025-03-20Results posted · ClinicalTrials.gov

A Randomized Phase II/III Trial of "Novel Therapeutics" Versus Azacitidine in Newly Diagnosed Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS), Age 60 or Older LEAP: Less-Intense AML Platform Trial

Clinical trial2025-01-16Results posted · ClinicalTrials.gov

Approval: Rydapt (EMA)

Regulatory2017-09-18EMA

Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial.

Research2017-07-24Leukemia

Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis.

Research2016-06-01The New England journal of medicine

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Midostaurin
Aliases & brands
4'-N-benzoylstaurosporineRydapt
RxNorm CUI
1919083
ChEMBL ID
CHEMBL608533
UNII
ID912S5VON
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2017-09-18
Latest approval
2017-09-18
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2017-09-18Approval
Approval: Rydapt (EMA)
Indication: Rydapt is indicated: in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response… Show full indication

Rydapt is indicated: in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive (see section 4.2); as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

37 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
37
registered trials across all phases
LATEST COMPLETION 2026
6
Active studies
2
Recruiting
7
Late-stage (III+)
18
Completed
13
Discontinued
PHASE DISTRIBUTIONn = 37
Phase 19Phase 1 / 24Phase 217Phase 2 / 31Phase 36

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3, Multicenter, Open-label, Randomized, 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 (AML) or Myelodysplastic Syndromes With Excess Blasts-2 (MDS-EB2) With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML / AMLSG 28-18)

Active, not recruitingPH IIIStichting Hemato-Oncologie voor Volwassenen Nederlandn=7772033

Phase III Randomized Study of Crenolanib Versus Midostaurin Administered Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed Subjects With FLT3 Mutated Acute Myeloid Leukemia

CompletedPH IIIArog Pharmaceuticals, Inc.n=2142026

A Phase 3, Prospective, Randomized Multi-center Intervention Trial of Early Intensification in AML Patients Bearing FLT3 Mutations Based on Peripheral Blast Clearance: A MYNERVA-GIMEMA Study

Unknown statusPH IIIGruppo Italiano Malattie EMatologiche dell'Adulton=1722025

A Randomized Phase II/III Trial of "Novel Therapeutics" Versus Azacitidine in Newly Diagnosed Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS), Age 60 or Older LEAP: Less-Intense AML Platform Trial

CompletedPH II/IIINational Cancer Institute (NCI)n=762024

An Open-label, Multi-Center, Phase IIIb Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia Who Are Eligible for "7+3" or "5+2" Chemotherapy

CompletedPH IIINovartis Pharmaceuticalsn=3012021

Recent completions

Trials that read out recently, adding to the completed evidence base.

Phase III Randomized Study of Crenolanib Versus Midostaurin Administered Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed Subjects With FLT3 Mutated Acute Myeloid Leukemia

CompletedPH IIIArog Pharmaceuticals, Inc.n=2142026

A Phase I Study to Evaluate the Safety and Tolerability of Gemtuzumab Ozogamicin and Midostaurin When Used in Combination With Standard Cytarabine and Daunorubicin Induction for Newly Diagnosed FLT3-mutated Acute Myeloid Leukemia

CompletedPH IUma Boraten=212025

A Randomized Phase II/III Trial of "Novel Therapeutics" Versus Azacitidine in Newly Diagnosed Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS), Age 60 or Older LEAP: Less-Intense AML Platform Trial

CompletedPH II/IIINational Cancer Institute (NCI)n=762024

V-FAST: A Phase 1b Master Trial to Investigate CPX-351 Combined With Various Targeted Agents in Subjects With Previously Untreated Acute Myeloid Leukemia

CompletedPH IJazz Pharmaceuticalsn=572023

A Phase II, Randomized, Double-blind, Multi-center, Placebo-controlled Study to Evaluate the Efficacy and Safety of Twice Daily Oral Midostaurin in Combination With Daunorubicin/Cytarabine Induction, High-dose Cytarabine Consolidation, and Midostaurin Single Agent Continuation Therapy in Newly Diagnosed Patients With FLT3-mutated Acute Myeloid Leukemia (AML).

CompletedPH IINovartis Pharmaceuticalsn=672022

An Open-label, Multi-Center, Phase IIIb Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia Who Are Eligible for "7+3" or "5+2" Chemotherapy

CompletedPH IIINovartis Pharmaceuticalsn=3012021

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20162018
Journals, researchers & institutions
Top journals
  • Leukemia1
  • The New England journal of medicine1
Leading researchers
  • George TI2
  • Gotlib J2
  • Akin C1
  • Awan FT1
  • Berube C1
  • Corless C1
  • Coutre S1
  • DeAngelo DJ1
Leading institutions
free-text, unnormalised
  • Dana-Farber Cancer Institute1
  • Massachusetts General Hospital1
  • Oregon Health and Sciences University1
  • Stanford University School of Medicine1
  • Stanford University School of Medicine-Stanford Cancer Institute1
  • Stanford University School of Medicine/Stanford Cancer Institute1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.