Midostaurin clinical trials
Midostaurin is named as an intervention in 5 current clinical trials in our Germany, Netherlands, Belgium, Switzerland, Ireland, France, Spain, Sweden, Austria, Norway, Australia, Italy, Poland, Czechia, Turkey, Japan, South Korea and Israel registers. 1 is recruiting patients now. The most common phase is Phase 2. The sponsor with the most trials is Stichting Hemato-Oncologie voor Volwassenen Nederland (2). The most-studied condition is Acute Myeloid Leukemia. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Midostaurin is approved
All regulators →Of the three regulators tracked here, the first to approve Midostaurin was the US, on 28 Apr 2017. It is approved in 3 of the 3 regulators tracked here.
- 2 EU orphan designations, most recently for treatment of mastocytosis (4 Aug 2010, positive)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Midostaurin patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Midostaurin, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 2 listed patents; the last listed expires 2 Dec 2030 (US 8575146).
- Rydapt for treatment of acute myeloid leukaemia: until 20 Sep 2027. Union Register
- Rydapt for treatment of mastocytosis: until 20 Sep 2027. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Midostaurin
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Midostaurin works
All drug targets →- Tyrosine-protein kinase receptor FLT3 inhibitor InhibitorTarget: FLT3 fms related receptor tyrosine kinase 3✓ In ChEMBL and the Guide to Pharmacology
- Stem cell growth factor receptor inhibitor InhibitorTarget: KIT KIT proto-oncogene, receptor tyrosine kinaseChEMBL only
- Platelet-derived growth factor receptor inhibitor InhibitorTarget: Platelet-derived growth factor receptorChEMBL only
- Protein kinase C (PKC) inhibitor InhibitorTarget: Protein kinase C (PKC)ChEMBL only
- Vascular endothelial growth factor receptor 2 inhibitor InhibitorTarget: KDR kinase insert domain receptorChEMBL only
- Myelodysplastic syndromePhase 3
- Aggressive systemic mastocytosisPhase 2
- Chronic myeloid leukemiaPhase 2
- Juvenile myelomonocytic leukemiaPhase 2
- LeukemiaPhase 2
- Myeloid leukemiaPhase 2
- Myeloid sarcomaPhase 2
- Systemic mastocytosis with an associated clonal hematologic non-mast cell lineage diseasePhase 2
- Acute leukemiaPhase 1/2
- Acute lymphoblastic leukemiaPhase 1/2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL608533, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 5702, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Midostaurin
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Midostaurin. 2 are current, and 2 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Midostaurin for treating advanced systemic mastocytosis NICE TA728 | Recommended | 22 Sep 2021 | Current |
| Midostaurin for untreated acute myeloid leukaemia NICE TA523 | Recommended | 13 Jun 2018 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Midostaurin
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: RydaptEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Midostaurin
- ATC code
- L01XE39
- ChEMBL
- CHEMBL608533
- DrugBank
- DB06595
- PubChem CID
- 9829523
- CAS number
- 120685-11-2
- FDA UNII
- ID912S5VON
- Wikidata
- Q6842945
- Wikipedia
- Midostaurin
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Midostaurin
5 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Midostaurin in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Midostaurin. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
Prospective evaluation of response to treatment by analysis of measurable residual disease in younger adult patients (18-60 years) with acute myeloid leukemia (AML). The Backbone InterGroup (BIG)-2 umbrella trial from the Acute Leukemia French Intergroup (ALFA, FILO) - BIG-2
2026-525759-96-00 · CTIS ↗ · Hemic and Lymphatic Diseases
|
Phase 3 | Authorised | Centre Hospitalier Universitaire De Toulouse | France |
Midostaurin across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
1 single-substance product containing Midostaurin on the HPRA medicines list.
- Rydapt 25 mg soft capsulesNovartis Europharm Limited · EU/1/17/1218/001
Supply shortages
No entry for Midostaurin in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Midostaurin
5 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Stichting Hemato-Oncologie voor Volwassenen Nederland | 2 |
| Novartis Pharmaceuticals | 1 |
| Technische Universität Dresden | 1 |
| Richard Stone, MD | 1 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Midostaurin trials
How many clinical trials are testing Midostaurin?
5 current trials in our Germany, Netherlands, Belgium, Switzerland, Ireland, France, Spain, Sweden, Austria, Norway, Australia, Italy, Poland, Czechia, Turkey, Japan, South Korea and Israel registers name Midostaurin (also registered as PKC412) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Midostaurin trials recruiting patients?
Yes. 1 of the 5 trials is recruiting: 1 with sites in Germany. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Midostaurin clinical trials?
Stichting Hemato-Oncologie voor Volwassenen Nederland (2), Novartis Pharmaceuticals (1), Technische Universität Dresden (1) and Richard Stone, MD (1). The number in brackets is how many trials each organisation leads.
Are there Midostaurin clinical trials in Ireland?
Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.
Is Midostaurin licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Midostaurin, for example Rydapt 25 mg soft capsules (Novartis Europharm Limited). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Midostaurin shortage?
Not in the lists we track: Midostaurin has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Midostaurin works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Midostaurin works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Midostaurin (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.