Midostaurin
Also known as Rydapt
NOVARTIS AG NVS NVSEF · Acute Myeloid Leukemia; Acute Myeloid Leukemia (AML)
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Approved
Not recorded
Linked trials
2
From ClinicalTrials.gov
Latest FDA action
Approved
NDA207997,
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- NOVARTIS AG NVS NVSEF Source: Company record for NOVARTIS AG (opens in a new tab)
- Indications
- Acute Myeloid Leukemia; Acute Myeloid Leukemia (AML) Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Application
- NDA 207997 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for NDA 207997 (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 Completed
- Phase 3 CompletedFirst trial start 13 Feb 20182 linked trials
- Filed Completed
- Approved Current stageFirst approval 28 Apr 2017
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
No catalysts yet. Editors add each date together with its source.
FDA designations
No FDA designations are on record for Midostaurin. Editors add each one with its source.
Filed application
No filed application is on record for Midostaurin. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for Midostaurin.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT03512197ClinicalTrials.gov record for NCT03512197 (opens in a new tab) | 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) | Phase 3 | Completed | 511 (actual) | 12 Feb 2021 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT03512197 (opens in a new tab) |
Posted results on ClinicalTrials.gov for NCT03512197
Serious adverse events (AEs were reported from 1st dose of study treatment until end of treatment plus 30 days, up to a maximum (max.) duration of 573 days (543 days max. exposure plus 30 days post-treatment) for midostaurin and up to a max. duration of 416 days (386 days max. exp. plus 30 days post-treatment) for placebo. Deaths - collected in the post-treatment survival follow-up period from 31 days after last dose of study medication until the end of the study, up to approx. 18 months. These are not considered AEs.): Midostaurin + Chemotherapy (On-treatment) 95 of 245; Placebo + Chemotherapy (On-treatment) 114 of 249; Midostaurin + Chemotherapy (Post-treatment Survival Follow-up) 0 of 0; Placebo + Chemotherapy (Post-treatment Survival Follow-up) 0 of 0 Source: ClinicalTrials.gov results for NCT03512197 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing NCT03512197 (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT03379727ClinicalTrials.gov record for NCT03379727 (opens in a new tab) | 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. | Phase 3 | Completed | 301 (actual) | 9 Jul 2021 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT03379727 (opens in a new tab) |
Posted results on ClinicalTrials.gov for NCT03379727 Serious adverse events (Adverse Events were reported from first dose of study treatment until end of treatment plus 30 days, up to a maximum duration of 25 months (24 months plus 1 month (30 days) post treatment).): Induction Phase 76 of 301; Consolidation Phase 67 of 210; Maintenance Phaase 7 of 93; Overall 137 of 301 Source: ClinicalTrials.gov results for NCT03379727 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing NCT03379727 (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
| Application | Action | Date | Outcome | Review | Source |
|---|---|---|---|---|---|
| NDA207997Action letter for NDA207997 (opens in a new tab) | Supplement 6 Efficacy | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA207997Action letter for NDA207997 (opens in a new tab) | Original 1 Efficacy | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA207997Action letter for NDA207997 (opens in a new tab) | Original 2 Type 1 - New Molecular Entity | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) |
Data provided by the U.S. Food and Drug Administration.
FDA recalls load here.
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Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
AuthorisedOrphan medicine
Rydapt (EMEA/H/C/004095)
- Authorised
- Therapeutic area
- Leukemia, Myeloid, Acute;Mastocytosis
- Marketing authorisation holder
- Novartis Europharm Ltd
- Active substance (INN)
- midostaurin
- ATC code
- L01XE
- Latest EMA decision
Matched by active substance: EMA's INN or active substance equals this drug's name.
EMA page for Rydapt (opens in a new tab)EMA medicine data (opens in a new tab)
Source: European Medicines Agency (EMA), medicines data download. Licence: EMA re-use with acknowledgement. EMA medicines report 2026-10-26.
Related news
No recent headlines mention this drug.
Sources
- Drugs@FDA 207997 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT03512197 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT03379727 (opens in a new tab) Licence: public-domain. Fetched
- Drugs@FDA via openFDA NDA207997 (opens in a new tab) Licence: cc0. Fetched
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.