Midostaurin

Other protein kinase inhibitors · given by oral · on the market since 2017

Leads

21.5M

People

Makers

How abandoned

What it's made of

Each functional group on the molecule does a specific, predictable job.

SETS WHERE IT GOES

Sugar ring

What it does. Extremely water-loving. Adding one to a molecule usually destroys its ability to cross the gut wall, which is why many such drugs must be injected.

What it opens up. If given by mouth it stays in the gut and acts there only — a targeting advantage for intestinal disease.

Where it can get to

Predicted from molecular weight, lipophilicity, polar surface area and hydrogen-bond donor count. A drug can only act where it distributes, so this constrains which conditions are addressable before any biology is considered. derived

Easily

Systemic circulation

Absorbed from the gut and distributed in plasma.

Barely

Central nervous system

Crosses the blood–brain barrier.

Intestinal lumen only

Poorly absorbed. Acts locally in the gut with minimal systemic exposure.

Barely

Skin

Crosses the stratum corneum. Viable as a topical.

Barely

Urinary tract

Cleared unchanged in urine. Local concentration exceeds plasma.

What it binds — and whether we can reach it · 1 of 1 reachable

Binding affinity is only meaningful against the plasma concentration reached at the approved dose. A target requiring higher concentrations cannot be engaged in a patient, whatever the in vitro potency. Most repurposing hypotheses fail at this step, and it is rarely checked before a trial is proposed. measured

Values are pAct — the negative logarithm of the molar concentration at which the drug half-occupies the target. Each whole step is a tenfold difference in potency: 9.0 = 1 nanomolar, 6.0 = 1 micromolar, 5.0 = 10 micromolar. Oral drugs typically reach 0.1–10 micromolar in plasma, so roughly 6.0 is the threshold below which a target cannot be reached at a tolerated dose. Figures come from DrugCentral’s curated binding data.

FLT3

Kinase · inhibitor

7.96

Engaged

Target	Kind	Action	Affinity (pAct)	Can it be engaged?
FLT3	Kinase	inhibitor	7.96	Engaged

New uses, and who is already trying them

One card per candidate indication: the reasoning link by link, the evidence tier that governs how strongly it can be stated, and any registered trials.

5 of the 59 conditions linked to this drug’s targets have been checked against trial records so far. The rest are not ruled out — they are unchecked.

Not yet used for these — 5

acute myeloid leukemia

Tried in people

It showed an effect in a human trial that was not followed up.

The condition

Acute myeloid leukemia (AML) is a group of neoplasms arising from precursor cells committed to the myeloid cell-line differentiation. All of them are characterized by clonal expansion of myeloid blasts. AML manifests by fever, pallor, anemia, hemorrhages and recurrent infections.

FLT3 · fms related receptor tyrosine kinase 3

Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine FLT3LG and regulates differentiation, proliferation and survival of hematopoietic progenitor cells and of dendritic cells. Promotes phosphorylation of SHC1 and AKT1, and activation of the downstream effector MTOR. Promotes activation of RAS signaling and phosphorylation of downstream kinases, including MAPK1/ERK2 and/or MAPK3/ERK1. Promotes phosphorylation of FES, FER, PTPN6/SHP, PTPN11/SHP-2, PLCG1, and STAT5A and/or STAT5B. Activation o

Open Targets scores each kind of evidence from 0.00 to 1.00 for this exact pairing — FLT3 and acute myeloid leukemia. The score reflects how much evidence of that kind exists and how consistent it is. It is not a probability that the drug works. Rows are ordered by how much each kind counts toward the evidence figure used in the ranking, not by its raw score, because the two disagree: literature co-occurrence saturates near 1.00 on almost any well-studied pair, while genetic association rarely exceeds 0.70 even when causal.

Genetic association0.55 / 1.00

Inherited variants in this gene alter disease risk in human populations. The datatype most predictive of later clinical success.55% of evidence score

Somatic mutation0.85 / 1.00

This gene is recurrently mutated in this tumour type.15% of evidence score

Animal model0.62 / 1.00

Altering this gene in animals reproduces or relieves the disease phenotype.15% of evidence score

Pathway0.84 / 1.00

The gene sits in a pathway with established disruption in this condition.8% of evidence score

Curated genetic literature0.30 / 1.00

Gene–disease links curated from published genetic studies.5% of evidence score

Literature co-occurrence1.00 / 1.00

Gene and condition appear together in published text. Reflects publication volume, not causal evidence.2% of evidence score

Clinical0.97 / 1.00

Derived from clinical or trial records.not counted

Phase 3 has run

A Phase 3 trial exists. Read its result before anything else.

NCT03258931 Study of Crenolanib vs Midostaurin Follo · Ph 3 · 214 people

+14 more on ClinicalTrials.gov

What would kill it. A null result in the completed Phase 3.

hepatocellular carcinoma

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition

A malignant tumor that arises from hepatocytes. Hepatocellular carcinoma is relatively rare in the United States but very common in all African countries south of the Sahara and in Southeast Asia. Most cases are seen in patients over the age of 50 years, but this tumor can also occur in younger individuals and even in children. Hepatocellular carcinoma is more common in males than females and is associated with hepatitis B, hepatitis C, chronic alcohol abuse and cirrhosis. Serum elevation of alpha-fetoprotein occur

FLT3 · fms related receptor tyrosine kinase 3

Open Targets scores each kind of evidence from 0.00 to 1.00 for this exact pairing — FLT3 and hepatocellular carcinoma. The score reflects how much evidence of that kind exists and how consistent it is. It is not a probability that the drug works. Rows are ordered by how much each kind counts toward the evidence figure used in the ranking, not by its raw score, because the two disagree: literature co-occurrence saturates near 1.00 on almost any well-studied pair, while genetic association rarely exceeds 0.70 even when causal.

Somatic mutation0.46 / 1.00

This gene is recurrently mutated in this tumour type.15% of evidence score

Literature co-occurrence0.48 / 1.00

Gene and condition appear together in published text. Reflects publication volume, not causal evidence.2% of evidence score

Clinical0.97 / 1.00

Derived from clinical or trial records.not counted

Nobody has tried this

No registered trial of this drug in this condition.

What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach FLT3 in the relevant tissue at tolerated doses.

gastrointestinal stromal tumor

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition

Gastrointestinal stromal tumor (GIST) is the most common mesenchymal neoplasm of the gastrointestinal (GI) tract, typically presenting in adults over the age of 40 (mean age 63), and only rarely in children, in various regions of the GI tract, most commonly the stomach or small intestine but also less commonly in the esophagus, appendix, rectum and colon. GISTs can be asymptomatic or present with various non-specific signs, depending on the location and size of tumor, such as loss of appetite, anemia, weight loss,

FLT3 · fms related receptor tyrosine kinase 3

Open Targets scores each kind of evidence from 0.00 to 1.00 for this exact pairing — FLT3 and gastrointestinal stromal tumor. The score reflects how much evidence of that kind exists and how consistent it is. It is not a probability that the drug works. Rows are ordered by how much each kind counts toward the evidence figure used in the ranking, not by its raw score, because the two disagree: literature co-occurrence saturates near 1.00 on almost any well-studied pair, while genetic association rarely exceeds 0.70 even when causal.

Somatic mutation0.46 / 1.00

This gene is recurrently mutated in this tumour type.15% of evidence score

Literature co-occurrence0.21 / 1.00

Gene and condition appear together in published text. Reflects publication volume, not causal evidence.2% of evidence score

Clinical0.95 / 1.00

Derived from clinical or trial records.not counted

Nobody has tried this

No registered trial of this drug in this condition.

What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach FLT3 in the relevant tissue at tolerated doses.

neoplasm

Tried in people

It showed an effect in a human trial that was not followed up.

The condition

A benign or malignant tissue growth resulting from uncontrolled cell proliferation. Benign neoplastic cells resemble normal cells without exhibiting significant cytologic atypia, while malignant cells exhibit overt signs such as dysplastic features, atypical mitotic figures, necrosis, nuclear pleomorphism, and anaplasia. Representative examples of benign neoplasms include papillomas, cystadenomas, and lipomas; malignant neoplasms include carcinomas, sarcomas, lymphomas, and leukemias.

FLT3 · fms related receptor tyrosine kinase 3

Open Targets scores each kind of evidence from 0.00 to 1.00 for this exact pairing — FLT3 and neoplasm. The score reflects how much evidence of that kind exists and how consistent it is. It is not a probability that the drug works. Rows are ordered by how much each kind counts toward the evidence figure used in the ranking, not by its raw score, because the two disagree: literature co-occurrence saturates near 1.00 on almost any well-studied pair, while genetic association rarely exceeds 0.70 even when causal.

Genetic association0.36 / 1.00

Inherited variants in this gene alter disease risk in human populations. The datatype most predictive of later clinical success.55% of evidence score

Literature co-occurrence0.81 / 1.00

Gene and condition appear together in published text. Reflects publication volume, not causal evidence.2% of evidence score

Clinical0.93 / 1.00

Derived from clinical or trial records.not counted

Phase 3 has run

A Phase 3 trial exists. Read its result before anything else.

NCT04027309 A Study of Gilteritinib Versus Midostaur · Ph 3 · 777 people

+14 more on ClinicalTrials.gov

What would kill it. A null result in the completed Phase 3.

renal cell carcinoma

Wide open

It acts on a target linked to this disease; untested, and the direction of effect is unverified.

The condition

A carcinoma that arises from glandular epithelial cells of the kidney

FLT3 · fms related receptor tyrosine kinase 3

Open Targets scores each kind of evidence from 0.00 to 1.00 for this exact pairing — FLT3 and renal cell carcinoma. The score reflects how much evidence of that kind exists and how consistent it is. It is not a probability that the drug works. Rows are ordered by how much each kind counts toward the evidence figure used in the ranking, not by its raw score, because the two disagree: literature co-occurrence saturates near 1.00 on almost any well-studied pair, while genetic association rarely exceeds 0.70 even when causal.

Somatic mutation0.30 / 1.00

This gene is recurrently mutated in this tumour type.15% of evidence score

Literature co-occurrence0.08 / 1.00

Gene and condition appear together in published text. Reflects publication volume, not causal evidence.2% of evidence score

Clinical0.98 / 1.00

Derived from clinical or trial records.not counted

Nobody has tried this

No registered trial of this drug in this condition.

What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach FLT3 in the relevant tissue at tolerated doses.

Why nobody will fund it

2 companies make this across 2 approved products. With that many manufacturers the price has fallen to production cost. A company funding a trial could not recover it — competitors could market the same molecule for the new indication immediately, without contributing.

How abandoned it is0 / 100

0 to 100, calculated from manufacturer count, remaining patent or exclusivity, and whether any reformulation route to exclusivity is still open. 100 means no route to commercial return exists. The weights behind it are unsourced.

THE ONE WAY A COMPANY COULD STILL OWN IT

injectableinhaledmodified-release

A new route or dosage form qualifies for a 505(b)(2) application, which carries three years of exclusivity for that formulation. It is the only remaining route to exclusivity on a generic molecule.

What it would cost to find out

Derived, not quoted. Events needed is 4(z_α+z_β)²/ln(HR)²; enrolment is that divided by the event rate and follow-up. The 1/ln(HR)² term dominates: halving the detectable effect size roughly quadruples enrolment. derived

Effect sought (hazard ratio)0.75

Lower = bigger effect = smaller trial

How often the event happens per year8.0%

Follow-up2 yr

Cost per patient$32K

Unsourced placeholder

379

Events needed

Patients

$93M

Trial cost

assumed

$12.30

Per person reached

assumed

Cost per person reached is the relevant metric for a non-commercial funder, since there is no revenue to set against the outlay.

Ask for ideas nobody has listed

Reasons from the functional groups and distribution above, excluding conditions already listed. Output is hypotheses for falsification, not findings. Not medical advice.

Who might pay for the trial

Trials of off-patent drugs are rarely funded by venture capital or pharma. They are paid for by government grants and disease foundations. Here is who has funded work on this molecule before, what federal mechanisms are open now, and which foundations fund trials in the disease areas it touches. measured

Federal grants that name this medicine

37 grants on NIH RePORTER

5R01CA258359-05(NCI · National Cancer Institute)Active

$360K (2026)

Inhibition of T-cell Receptor Signaling for Treatment of Adult T-cell Leukemia Lymphoma

PI: Ratner, Lee · Washington University, Saint Louis, MO · through 2027-06

View on NIH RePORTER ↗Apply to same mechanism (PAR-21-033) ↗

1P50CA298970-01A1(NCI · National Cancer Institute)Active

$502K (2026)

Project 3: Targeting mSWI/SNF in AML

PI: Kadoch, Cigall · Dana-Farber Cancer Inst, Boston, MA · through 2031-07

View on NIH RePORTER ↗Apply to same mechanism (PAR-23-284) ↗

5R01CA267978-05(NCI · National Cancer Institute)Active

$446K (2026)

3H-pyrazolo[4,3-f]quinoline-containing compounds as selective and tunable protein kinase inhibitors

PI: Sintim, Herman O · University Of Notre Dame, Notre Dame, IN · through 2027-04

View on NIH RePORTER ↗Apply to same mechanism (PA-20-185) ↗

See all 37 projects on NIH RePORTER →

Open federal programmes that would fund this work

Open notices with an explicit mandate to fund work on approved or off-patent molecules.

RFA-CA-25-033National Institutes of HealthForecast — not open yet

Glioblastoma Therapeutics Network (GTN; U19 Clinical Trial Required)

Grants.gov (RFA-CA-25-033) ↗Direct listing ↗Who already holds this grant ↗

RFA-FD-25-020Food and Drug Administrationcloses 05/16/2028

Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet

Read announcement ↗Grants.gov (RFA-FD-25-020) ↗Direct listing ↗Who already holds this grant ↗

FOR-FD-25-020Food and Drug AdministrationForecast — not open yet

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R

Grants.gov (FOR-FD-25-020) ↗Direct listing ↗Who already holds this grant ↗

FOR-FD-25-001Food and Drug AdministrationForecast — not open yet

Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01

Grants.gov (FOR-FD-25-001) ↗Direct listing ↗Who already holds this grant ↗

RFA-TR-25-002National Institutes of Healthcloses 07/02/2027

Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not

Read announcement ↗Grants.gov (RFA-TR-25-002) ↗Direct listing ↗Who already holds this grant ↗

PAR-25-374National Institutes of Healthcloses 05/07/2028

Translational Bioinformatics and Experimental Approaches to Advance Drug Repos

Read announcement ↗Grants.gov (PAR-25-374) ↗Direct listing ↗Who already holds this grant ↗

HT942526PRCRPCTADefense Health Agency Contracting Activity -closes 10/05/2026

DoW Peer Reviewed Cancer, Clinical Trial Award

Grants.gov (HT942526PRCRPCTA) ↗Direct listing ↗Who already holds this grant ↗

PAR-26-049National Institutes of HealthForecast — not open yet

Establishing Pediatric CNS Pharmacodynamic Measures as Tools to Enable Psychia

Grants.gov (PAR-26-049) ↗Direct listing ↗Who already holds this grant ↗

Showing 8 of 990 currently open under this category on Grants.gov, matched to the disease areas this drug touches. derived Browse all 990 on Grants.gov ↗

Funders beyond the federal system

Grants.gov lists US federal money. Many off-patent drug trials are supported by disease foundations and nonprofits. These are matched to the body systems this drug touches. assumed

Matched by disease area, not by molecule — a foundation appearing here funds work in a field this drug touches.

Who manufactures it

Companies holding an approved abbreviated application for this molecule in the United States, from the FDA Orange Book. Supply already exists at scale, so a trial would not need a manufacturing programme — but any of these could also be a partner, or an objector.

Dr Reddys Laboratories LtdLupin Inc

Manufacturers

companies with their own approval

Approved products

distinct strengths and forms

Routes

oral

2017

Oldest product still sold

approval year of the earliest product still on the market

ALSO SOLD AS

RYDAPT

Nothing on this page is medical advice. Candidate uses are unproven hypotheses derived from public data; established uses are listed for verification only. Figures carry their provenance inline — anything marked assumed or derived is not a measurement.

← Back to the full ledger

What it's made of

Sugar ring

Where it can get to

What it binds — and whether we can reach it · 1 of 1 reachable

New uses, and who is already trying them

acute myeloid leukemia

hepatocellular carcinoma

gastrointestinal stromal tumor

neoplasm

renal cell carcinoma

Why nobody will fund it

What it would cost to find out

Ask for ideas nobody has listed

Who might pay for the trial

Who manufactures it

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