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Ethambutol Hydrochloride

Other drugs for treatment of tuberculosis · Combinations of drugs for treatment of tuberculosis · given by oral · on the market since 1999

5
Leads
35.2M
People
2
Makers
9
How abandoned
01

What it's made of

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

SETS WHERE IT GOES

Basic amine

What it does. Neutral enough to slip through membranes, then becomes charged inside the acidic compartments of the cell and gets trapped there. Concentrations inside cells can reach many times the blood level.

What it opens up. Diseases of the cell's internal recycling compartments, and a common reason a drug accumulates in lung, liver, and eye tissue far beyond what the blood level suggests.

02

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.
Partly
Central nervous system
Crosses the blood–brain barrier.
No
Intestinal lumen only
Poorly absorbed. Acts locally in the gut with minimal systemic exposure.
Partly
Skin
Crosses the stratum corneum. Viable as a topical.
Easily
Urinary tract
Cleared unchanged in urine. Local concentration exceeds plasma.
03

What it binds — and whether we can reach it · 1 of 5 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.
CYP3A4
Enzyme ·
6.25
Just reachable
HTR2A
GPCR ·
5.48
Out of reach
HTR2C
GPCR ·
5.16
Out of reach
HTR2B
GPCR ·
5.15
Out of reach
CYP3A5
Enzyme ·
4.72
Not reachable

This molecule builds up inside cells far above blood levels, concentrates in urine. Local concentration therefore exceeds plasma, so targets that look unreachable on a plasma reading may still be engaged. assumed

04

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 57 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

major depressive disorder

Wide open

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

The condition
An episode of depression lasting two or more weeks without an intervening episode of mania.
HTR2A · 5-hydroxytryptamine receptor 2A
G protein-coupled receptor for 5-hydroxytryptamine (serotonin) (PubMed:1330647, PubMed:18703043, PubMed:19057895, PubMed:21645528, PubMed:22300836, PubMed:35084960, PubMed:38552625). Also functions as a receptor for various drugs and psychoactive substances, including mescaline, psilocybin, 1-(2,5-dimethoxy-4- iodophenyl)-2-aminopropane (DOI) and lysergic acid diethylamide (LSD) (PubMed:28129538, PubMed:35084960). Ligand binding causes a conformation change that triggers signaling via guanine nucleotide- binding pr
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 HTR2A in the relevant tissue at tolerated doses.

schizophrenia

Wide open

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

The condition
A major psychotic disorder characterized by abnormalities in the perception or expression of reality. It affects the cognitive and psychomotor functions. Common clinical signs and symptoms include delusions, hallucinations, disorganized thinking, and retreat from reality.
HTR2A · 5-hydroxytryptamine receptor 2A
G protein-coupled receptor for 5-hydroxytryptamine (serotonin) (PubMed:1330647, PubMed:18703043, PubMed:19057895, PubMed:21645528, PubMed:22300836, PubMed:35084960, PubMed:38552625). Also functions as a receptor for various drugs and psychoactive substances, including mescaline, psilocybin, 1-(2,5-dimethoxy-4- iodophenyl)-2-aminopropane (DOI) and lysergic acid diethylamide (LSD) (PubMed:28129538, PubMed:35084960). Ligand binding causes a conformation change that triggers signaling via guanine nucleotide- binding pr
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 HTR2A in the relevant tissue at tolerated doses.

autism

Wide open

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

The condition
Persistent deficits in social interaction and communication and interaction as well as a markedly restricted repertoire of activity and interest as well as repetitive patterns of behavior.
HTR2A · 5-hydroxytryptamine receptor 2A
G protein-coupled receptor for 5-hydroxytryptamine (serotonin) (PubMed:1330647, PubMed:18703043, PubMed:19057895, PubMed:21645528, PubMed:22300836, PubMed:35084960, PubMed:38552625). Also functions as a receptor for various drugs and psychoactive substances, including mescaline, psilocybin, 1-(2,5-dimethoxy-4- iodophenyl)-2-aminopropane (DOI) and lysergic acid diethylamide (LSD) (PubMed:28129538, PubMed:35084960). Ligand binding causes a conformation change that triggers signaling via guanine nucleotide- binding pr
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 HTR2A in the relevant tissue at tolerated doses.

psychotic disorder

Wide open

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

The condition
An abnormal condition of the mind that involves a loss of contact with reality. People experiencing psychosis may exhibit personality changes and thought disorder. Depending on its severity, this may be accompanied by unusual or bizarre behavior, as well as difficulty with social interaction and impairment in carrying out daily life activities.
HTR2A · 5-hydroxytryptamine receptor 2A
G protein-coupled receptor for 5-hydroxytryptamine (serotonin) (PubMed:1330647, PubMed:18703043, PubMed:19057895, PubMed:21645528, PubMed:22300836, PubMed:35084960, PubMed:38552625). Also functions as a receptor for various drugs and psychoactive substances, including mescaline, psilocybin, 1-(2,5-dimethoxy-4- iodophenyl)-2-aminopropane (DOI) and lysergic acid diethylamide (LSD) (PubMed:28129538, PubMed:35084960). Ligand binding causes a conformation change that triggers signaling via guanine nucleotide- binding pr
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 HTR2A in the relevant tissue at tolerated doses.

bipolar disorder

Wide open

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

The condition
A disorder of the brain that causes unusual shifts in mood, energy, activity levels and the ability to carry out day-to-day tasks. Often these moods range and shift from periods of elation and energized behavior to those of hopelessness and depression.
HTR2A · 5-hydroxytryptamine receptor 2A
G protein-coupled receptor for 5-hydroxytryptamine (serotonin) (PubMed:1330647, PubMed:18703043, PubMed:19057895, PubMed:21645528, PubMed:22300836, PubMed:35084960, PubMed:38552625). Also functions as a receptor for various drugs and psychoactive substances, including mescaline, psilocybin, 1-(2,5-dimethoxy-4- iodophenyl)-2-aminopropane (DOI) and lysergic acid diethylamide (LSD) (PubMed:28129538, PubMed:35084960). Ligand binding causes a conformation change that triggers signaling via guanine nucleotide- binding pr
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 HTR2A in the relevant tissue at tolerated doses.
05

Why nobody will fund it

2 companies make this across 4 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 is9 / 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
topicalinjectableinhaledmodified-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.

06

What it would cost to find out

Derived, not quoted. Events needed is 4(zα+zβ)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

0.75
Lower = bigger effect = smaller trial
8.0%
2 yr
$32K
Unsourced placeholder
379
Events needed
3K
Patients
$93M
Trial cost
assumed
$7.53
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.

07

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.

08

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
340 grants on NIH RePORTER
1R21AI201981-01(NIAID · National Institute of Allergy & Infectious Diseases)Active
$233K (2026)
Cethromycin-based treatment of M. avium disease
PI: Sullivan, David Joseph · Johns Hopkins University, Baltimore, MD · through 2028-06
5R01AI183405-03(NIAID · National Institute of Allergy & Infectious Diseases)Active
$132K (2026)
Treatment of isoniazid-resistant tuberculosis: closing evidence gaps on safety, effectiveness, and pharmacokinetics of the standard regimen
PI: Mendoza, Alberto · Socios En Salud Sucursal Peru, Lima · through 2029-05
5R01AI179827-03(NIAID · National Institute of Allergy & Infectious Diseases)Active
$566K (2026)
Short course combination regimens for treatment of Mycobacterium avium pulmonary disease: a translational bench-to-bedside approach
PI: Kant, Shashi · University Of Texas Hlth Ctr At Tyler, Tyler, TX · through 2028-10
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)
RFA-FD-25-020Food and Drug Administrationcloses 05/16/2028
Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet
FOR-FD-25-020Food and Drug AdministrationForecast — not open yet
Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R
FOR-FD-25-001Food and Drug AdministrationForecast — not open yet
Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01
RFA-TR-25-002National Institutes of Healthcloses 07/02/2027
Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not
PAR-25-374National Institutes of Healthcloses 05/07/2028
Translational Bioinformatics and Experimental Approaches to Advance Drug Repos
HT942526PRCRPCTADefense Health Agency Contracting Activity -closes 10/05/2026
DoW Peer Reviewed Cancer, Clinical Trial Award
PAR-26-049National Institutes of HealthForecast — not open yet
Establishing Pediatric CNS Pharmacodynamic Measures as Tools to Enable Psychia
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.
09

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.

Epic Pharma LlcLupin Ltd
2
Manufacturers
companies with their own approval
4
Approved products
distinct strengths and forms
1
Routes
oral
1999
Oldest product still sold
approval year of the earliest product still on the market
ALSO SOLD AS
MYAMBUTOL

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.

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