Midostaurin
Small molecule targets FLT3, KDR, KIT via inhibition
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed acute megakaryoblastic leukemia in adult 2 milestones | |||||
| Filed | Other | — | acute megakaryoblastic leukemia in adult | 2016-10-25 | “PKC412 (midostaurin) was filed with the EMA and Swissmedic for the treatment of newly diagnosed FLT3 mutation-positive acute myeloid leukemia and advanced systemic mastocytosis.” novartis.com ↗ |
| Filed | EU (EMA) | — | acute megakaryoblastic leukemia in adult | 2016-10-25 | “PKC412 (midostaurin) was filed with the EMA and Swissmedic for the treatment of newly diagnosed FLT3 mutation-positive acute myeloid leukemia and advanced systemic mastocytosis.” novartis.com ↗ |
| Filed systemic mastocytosis 2 milestones | |||||
| Filed | Other | — | systemic mastocytosis | 2016-10-25 | “PKC412 (midostaurin) was filed with the EMA and Swissmedic for the treatment of newly diagnosed FLT3 mutation-positive acute myeloid leukemia and advanced systemic mastocytosis.” novartis.com ↗ |
| Filed | EU (EMA) | — | systemic mastocytosis | 2016-10-25 | “PKC412 (midostaurin) was filed with the EMA and Swissmedic for the treatment of newly diagnosed FLT3 mutation-positive acute myeloid leukemia and advanced systemic mastocytosis.” novartis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| 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
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2026-03-09 | Astellas Pharma Inc | Results Astellas and Hovon Confirm Phase 3 Study Did not Meet its Primary Endpoint of Overall Survival in Patients with Newly Diagnosed FLT3 m+ AML astellas.com ↗
While the study did not meet its primary endpoint of OS, gilteritinib showed a comparable OS benefit to midostaurin-based treatment in newly diagnosed FLT3 m+ AML. |
| 2022-11-03 | Jazz Pharmaceuticals | Results Jazz Pharmaceuticals to Present Data at 2022 ASH Meeting Showcasing Commitment to Advancing Oncology Research jazzpharma.com ↗
Preliminary results suggest the combination of Vyxeos + MID is feasible with a manageable safety profile and promising remission rates in adults with newly diagnosed, FLT3 -mutated AML. |
| 2017-09-20 | Novartis Pharmaceuticals | Regulatory 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 ↗
Novartis today announced that the European Commission (EC) approved Rydapt ® (midostaurin) for two indications in rare, hard-to-treat cancers. |
| 2017-07-21 | Novartis Pharmaceuticals | Regulatory 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 ↗
We are pleased with the positive recommendation from the CHMP and excited to move a step closer to bringing this much-needed treatment to these patients across Europe. |
| 2017-06-23 | Novartis Pharmaceuticals | Results NEJM publishes full analysis of Rydapt® (midostaurin) Phase III RATIFY trial in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) novartis.com ↗
Novartis today announced that full results from the Rydapt [®] (midostaurin) Phase III RATIFY (CALGB 10603 [Alliance]) clinical trial were published in The New England Journal of Medicine (NEJM) [1]. |
| 2017-04-28 | Novartis Pharmaceuticals | Regulatory Novartis receives FDA approval for Rydapt® in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of systemic mastocytosis (SM) novartis.com ↗
Novartis announced today the US Food and Drug Administration (FDA) has approved Rydapt ® (midostaurin, formerly PKC412) for two indications. |
| 2016-11-14 | Novartis Pharmaceuticals | Regulatory 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 | Results Novartis PKC412 (midostaurin) pivotal data published in NEJM show 60% response rate in advanced systemic mastocytosis (SM) novartis.com ↗
In the largest and longest-running prospective trial in advanced SM, 60% of patients achieved complete or partial resolution of organ damage[1] |
| 2016-02-19 | Novartis Pharmaceuticals | Regulatory Novartis drug PKC412 (midostaurin) receives Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute myeloid leukemia (AML) novartis.com ↗
Novartis announced today that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to PKC412 (midostaurin). |
| 2015-12-06 | Novartis Pharmaceuticals | Results Novartis drug PKC412 (midostaurin) improves overall survival by 23% in global Phase III study of AML patients with FLT3 mutations novartis.com ↗
Novartis today announced positive results from the global Phase III RATIFY (CALGB 10603) clinical trial. |
| 2015-11-30 | Novartis Pharmaceuticals | Results Novartis highlights clinical advances at ASH 2015, underscoring leadership in hematology research novartis.com ↗
The Multi-Kinase Inhibitor Midostaurin (M) Prolongs Survival Compared with Placebo (P) |
All press releases naming this drug 14 releases
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 ↗ |