Drugs / Midostaurin
Midostaurin
also known as
PKC412 · CGP 41251 · Midostaurina · Midostaurine · N-benzoylstaurosporine · NSC-656576 · NVP-PKC412 · Rydapt
Small molecule targets FLT3, KDR, KIT via inhibition
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | RYDAPT | — | 2020-11-24 | fda.gov ↗ |
| Approved | EU (EMA) | Rydapt | — | 2017-09-18 | europa.eu ↗ |
| Approved | US (FDA) | RYDAPT | — | 2017-04-28 | fda.gov ↗ |
Trials 37 · a red edge is where a trial was stopped
Also used as a comparator or background therapy in 12 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 14 trials | ||||||
| Phase 1 | NCT06313437 Background | Dec 2024 → Mar 2027 expected | acute myeloid leukemia | Dana-Farber Cancer Institute | Recruiting | No outcome recorded |
| Phase 1 | NCT03900949 Background | Mar 2019 → Aug 2024 | acute myeloid leukemia | Uma Borate | Completed | No outcome recorded |
| Phase 1 | NCT02078609 Background | Mar 2014 → Apr 2019 | acute myeloid leukemia | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT00819546 Background | Jan 2009 → Dec 2026 expected | acute myeloid leukemia, myelodysplastic syndrome | Dana-Farber Cancer Institute | Active not recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03760445 Background | Nov 2019 → Jul 2021 | acute myeloid leukemia | Novartis Pharmaceuticals | Withdrawn | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT03836209 Comparator | Dec 2019 → Oct 2023 overdue | acute myeloid leukemia | PrECOG, LLC. | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02115295 Background | May 2014 → May 2030 expected | acute biphenotypic leukemia, blast phase chronic myelogenous leukemia, BCR-ABL1 positive, myelodysplastic syndrome | M.D. Anderson Cancer Center | Recruiting | No outcome recorded |
| Phase 35 trials | ||||||
| Phase 3 | NCT04676243 Comparator | May 2022 → May 2025 | acute myeloid leukemia | University Hospital Heidelberg | Withdrawn | No outcome recorded |
| Phase 3 | NCT04174612 Background | Apr 2020 → Dec 2022 | acute myeloid leukemia, FLT3 internal tandem duplication | GIMEMA Foundation | Unknown | No outcome recorded |
| Phase 3 | NCT04027309 Comparator | Dec 2019 → Oct 2025 overdue | acute myeloid leukemia, myelodysplastic syndrome with excess blasts-2 | HOVON Foundation | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03258931 Comparator | Aug 2018 → Apr 2026 | acute myeloid leukemia | Arog Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT02665065 Comparator | Jun 2016 → Jun 2022 overdue | acute myeloid leukemia | Actinium Pharmaceuticals | Active not recruiting | No outcome recorded |
Press releases naming this drug 8 releases
| Date | Issuer | Release |
|---|---|---|
| 2017-09-20 | Novartis Pharmaceuticals | Novartis drug Rydapt® (midostaurin) receives EU approval for newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of advanced systemic mastocytosis (SM) novartis.com ↗ |
| 2017-07-21 | Novartis Pharmaceuticals | Novartis receives positive CHMP opinion for Rydapt® (midostaurin) for newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of advanced systemic mastocytosis (SM) novartis.com ↗ |
| 2017-06-23 | Novartis Pharmaceuticals | NEJM publishes full analysis of Rydapt® (midostaurin) Phase III RATIFY trial in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) novartis.com ↗ |
| 2017-04-28 | Novartis Pharmaceuticals | Novartis receives FDA approval for Rydapt® in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of systemic mastocytosis (SM) novartis.com ↗ |
| 2016-11-14 | Novartis Pharmaceuticals | Novartis drug PKC412 (midostaurin) granted FDA Priority Review for newly-diagnosed FLT3-mutated AML and advanced systemic mastocytosis novartis.com ↗ |
| 2016-06-29 | Novartis Pharmaceuticals | Novartis PKC412 (midostaurin) pivotal data published in NEJM show 60% response rate in advanced systemic mastocytosis (SM) novartis.com ↗ |
| 2016-02-19 | Novartis Pharmaceuticals | Novartis drug PKC412 (midostaurin) receives Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute myeloid leukemia (AML) novartis.com ↗ |
| 2015-12-06 | Novartis Pharmaceuticals | Novartis drug PKC412 (midostaurin) improves overall survival by 23% in global Phase III study of AML patients with FLT3 mutations novartis.com ↗ |
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 52 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Midostaurin | “Usually daunorubicin/ cytarabine or idarubicin/Cytarabine plus FLT3 inhibitor (usually midostaurin)” NCT04676243 ↗45“Midostaurin (for FLT3 mutant or FLT3-ITD subjects only, not allowed as single agent)” NCT02665065 ↗ “Local SOC is "7+3" + Midostaurin (100 mg) or Quizartinib (35.4 mg)” NCT07425808 ↗ |
| Known as | CGP 41251 | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | Midostaurina | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | Midostaurine | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | N-benzoylstaurosporine | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | NSC-656576 | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | NVP-PKC412 | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Known as | PKC412 | “An Expanded Treatment Protocol (ETP) of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia (AML)” NCT03114228 ↗7“This study combines midostaurin (PKC412) with radiation and a standard chemotherapy drug call 5-Fluorouracil (5-FU) for subjects with advanced rectal cancer.” NCT01282502 ↗ “PKC412 (midostaurin) is known to inhibit the c-KIT RTK activity as well as the FLT3 kinase, both in patients with ITD and TKD mutations.” NCT01830361 ↗ “midostaurin (also known as PKC412)” NCT00651261 ↗ “Midostaurin (PKC412)” NCT00782067 ↗ |
| Known as | Rydapt | ChEMBL registry synonym — accepted as the source's own label CHEMBL608533 ↗ |
| Action | Inhibit | “Vascular endothelial growth factor receptor 2 inhibitor” CHEMBL608533 ↗1“FLT3 kinase inhibitor midostaurin” PMID 22627678 ↗ Apr 2012 |
| Modality | Small molecule | “midostaurin - an oral multitargeted kinase inhibitor that is active in patients with a FLT3 mutation” PMID 28644114 ↗ Jun 2017 |
| Route | Oral | “Participants received 75 mg PKC412 three time daily (tid) orally” NCT00045942 ↗ |
| Target | FLT3 | “FLT3 kinase inhibitor midostaurin” PMID 22627678 ↗ Apr 2012 |
| Target | KDR | “Vascular endothelial growth factor receptor 2 inhibitor” CHEMBL608533 ↗ |