This page shows what was measured, who it was measured in, and what that does not settle.
What Osilodrostat does in the body
Endogenous hypercortisolemia
From the FDA-approved label: Osilodrostat is a cortisol synthesis inhibitor. It inhibits 11beta-hydroxylase (CYP11B1), the enzyme responsible for the final step of cortisol biosynthesis in the adrenal gland. 1 nM (n = 4).
What happened in people
RNAWiki has not yet published a reviewed conclusion for this use.
§ A fixed RNAWiki sentence
Where this came from
Wording RNAWiki always uses, not a finding about this substance.
No reviewed claim names a result for any goal on this record.
No source is stored against this line.
The limit that matters most
Not recorded.
Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
Supervision
RNAWiki has not recorded a supervision or regulatory status for this substance.
What the registries record it as
The substance registry classes this as chemical.
FDA substance registry · Y6581YAW9V · read 2026-08-29
Where each sentence above came from
The recorded explanation is written in label language rather than for a beginner. The page opens with what it is taken for instead, and the recorded explanation follows below.
Shown as the opening line on this page.
No statement of the main limit is recorded.
The four opening statements run to 47 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Stand-in result
A stand-in result is a number measured because the real result takes too long.
A picture of it, and where the picture fails
It is like judging a journey by the speedometer rather than by arriving.
Where that stops being true. Speed does predict arrival. Many stand-in results do not predict the real one.
What people get wrong. A stand-in result is often reported as the result itself.
A surrogate endpoint substituted for a clinical endpoint, valid only where the substitution has been shown to hold.
Enzyme
An enzyme is a protein that speeds up one chemical change.
A picture of it, and where the picture fails
An enzyme is like a machine on a production line doing one cut.
Where that stops being true. A machine is switched on and off by a person. Enzymes are controlled by the cell.
What people get wrong. Enzymes are thought to be used up. They are not; they work again and again.
A catalytic protein that lowers the activation energy of a specific reaction.
Receptor
A receptor is a part of a cell that a signal fits into.
A picture of it, and where the picture fails
A receptor is like a lock waiting for one key.
Where that stops being true. A lock either opens or does not. A receptor can be half-triggered, or worn out.
What people get wrong. Fitting a receptor is read as causing a benefit. It causes a step, and nothing more.
A protein that binds a specific ligand and converts that binding into a cellular response.
Pathway
A pathway is a chain of steps inside a cell, each one setting off the next.
A picture of it, and where the picture fails
A pathway is like a row of dominoes.
Where that stops being true. Dominoes fall one way. Pathways loop back and can switch themselves off.
What people get wrong. Changing one step is read as changing the outcome. Other steps often absorb it.
An ordered series of molecular interactions producing a defined cellular change.
What happened in people◇Read from sources, not yet reviewed
What was measured, goal by goal
One row for each goal a registered study measured something for. One column for each kind of thing that could be measured.
Registered studies list 6 outcome measures that RNAWiki could read. A registered study says what someone planned to measure. It does not say what they found. 0 of the matched studies tested this substance, and 0 posted a result.
There is no single score. A strong test result and a weak life result are different facts.
Goals down the side, kinds of measurement across the top. Each cell says what kind of thing was registered, not what was found.
Goal
Life outcome
What a body can do
How a person feels
A test result
A step in the body
Harms
How long
Who was studied
Blood sugar
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
△Only a number moved1 registered test measure.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Body weight
∅Nothing in the sources checkedNo registered study lists a life outcome for this goal.
∅Nothing in the sources checkedNo registered study lists a performance measure for this goal.
∅Nothing in the sources checkedNo registered study lists a symptom measure for this goal.
△Only a number moved1 registered test measure.
∅Nothing in the sources checkedNo registered study lists a body-step measure for this goal.
—Not recordedHarms were not a registered measure for this goal.
—Not recordedNo finished study window is recorded.
…Waiting for a reviewerWho was studied is listed further down the page.
Which registered measures put each goal on this table
Blood sugar
fasting glucose hba1c
Body weight
body composition
Sorted by fixed word lists, version v1. A name the rules do not recognise stays unsorted rather than moving to the nearest column.
What each mark on this table means
∅ Nothing in the sources checked
No registered study lists a life outcome for this goal.
△ Only a number moved
1 registered test measure.
— Not recorded
Harms were not a registered measure for this goal.
… Waiting for a reviewer
Who was studied is listed further down the page.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
Core Period: Change From Baseline in Mean Sitting Diastolic Blood Pressure (MSDBP) at Week 8 Last Observation Carried Forward (LOCF), as Measured by Office Blood Pressure (OBP)
✗ The study did not show it
Who was studied
NCT00758524
How many people
628
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Change From Baseline in MSSBP at Week 8 Last Observation Carried Forward (LOCF)
✗ The study did not show it
Who was studied
NCT00817635
How many people
155
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Comparison of the efficacy of metyrapone and osilodrostat
✗ The study did not show it
Who was studied
NCT07268222
How many people
150
Study design
Phase 4
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Percentage of Primary Efficacy Responder at Week 34 by Randomized Treatment and Strata
✗ The study did not show it
Who was studied
NCT02180217
How many people
137
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Number of Participants With Adverse/Serious Adverse Events
✗ The study did not show it
Who was studied
NCT03606408
How many people
127
Study design
Phase 2
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Percentage of Randomized Participants With a Complete Response
✗ The study did not show it
Who was studied
NCT02697734
How many people
73
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What happened in people◇Read from sources, not yet reviewed
How close this is to real life
The top step is something a person would feel or care about. The bottom is a guess from software. Filled steps are where evidence exists for this substance.
□Living longer, or avoiding a major eventNo evidence recorded. Death, a heart attack, a stroke, a hospital stay.No registered study measures this.
□What a body can do day to dayNo evidence recorded. Walking, dressing, breathing, recovering.No registered study measures this.
□Measured performanceNo evidence recorded. How much was lifted, how far was run, how fast.No registered study measures this.
□Symptoms and quality of lifeNo evidence recorded. Pain, tiredness, mood, sleep, as the person rated it.No registered study measures this.
■A number that stands in for healthEvidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.3 registered measures of this kind.
■A step measured inside a personEvidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
■AnimalsEvidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.Stored records in Rat. A result in animals says what to test next. It does not say what happens in people.
□Cells in a dishNo evidence recorded. Cells or chemistry on a bench, far from a whole body.No cell or bench record is stored.
□A guess from softwareNo evidence recorded. Nobody measured it. RNAWiki never publishes one as a finding.RNAWiki stores no prediction for this record. Software can suggest a link. RNAWiki does not publish one as a finding.
Higher on these steps means closer to something a person would feel. It does not mean better done.
What it would be like to take◇Read from sources, not yet reviewed
Felt, measured, or meaningful
Three different things that get called the same word. Registered studies measured all three, and mixing them is how a blood test becomes a health claim.
Felt
Things a person could notice without a test.
No registered study measured anything of this kind.
Measured
Things only a test, a scale or a device shows.
fasting glucose hba1c
bone mineral density
body composition
Meaningful
Things that change how a life goes, not only a number.
No registered study measured anything of this kind.
A registered study says what someone planned to measure. It does not say what they found. A number moving is not the same as a life going better. The two are shown apart here.
Felt, but not shown to help. A person notices a change. No study showed it leads anywhere good.
Measured, but not felt. A number moves. The person notices nothing. Both can be true.
Measured, but not shown to matter. A number moves in the good direction. Nobody showed that lives went better.
Matters, but takes years. The result that counts may take longer than anyone would keep watching.
Names that fit none of the three (3)
adverse events
adrenal insufficiency
comparison of the efficacy of metyrapone and osilodristat
These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.
What it would be like to take◇Read from sources, not yet reviewed
How long anything takes
Nine different lengths of time that get confused with each other. None of them is worked out from another.
Before anything is noticed.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before a test result moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
Before performance moves.RNAWiki does not store this separately, and never works it out from another figure on this page.
How long the result was watched.No finished study window is recorded for a study that tested this substance.
How long people took it.How long people actually took it is not stored. The study window is not the same thing.
How long people were followed.Follow-up length is not stored separately. It is never read off the study window, which would be a different thing.
How fast the body clears it. 4 hours hours
Read from the label, which states: “Elimination The elimination half-life of osilodrostat is approximately 4 hours.”
How long effects linger.RNAWiki does not store this separately, and never works it out from another figure on this page.
Beyond the studies. Nothing is recorded about the long term.
The longest finished study sets the edge of what anyone measured.
A study window is not how long people took it, and neither is how long they were followed. Where RNAWiki holds only one of the three, it shows one.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
ISTURISA is indicated for the treatment of endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is not an option or has not been curative. ISTURISA is a cortisol synthesis inhibitor indicated for the treatment of endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is not an option or has not been curative ( 1 )
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “The safety and effectiveness of ISTURISA in pediatric patients have not been established.”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
On older people, the label states: “Of the 167 patients in clinical trials with ISTURISA, 10 (6%) were 65 years and older.”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no available data on osilodrostat use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no available data on the presence of osilodrostat in human or animal milk, the effects on the breastfed infant, or the effects on milk production.”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
On people with reduced liver function, the label states: “Dosage adjustment is not required in patients with mild hepatic impairment (Child-Pugh A) but is required for patients with moderately impaired hepatic function (Child-Pugh B) and for patients with severe hepatic impairment (Child-Pugh C) [see Dosage and Administration (2.3) , Clinical Pharmacology (12.3) ] .”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
On people with reduced kidney function, the label states: “No dosage adjustment of ISTURISA in patients with impaired renal function is required [see Dosage and Administration (2.4) , Clinical Pharmacology (12.3) ] .”
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
Where the result stopped carrying
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied for a different goal
The studies measured something else entirely.
On this record: Some registered studies measured things that match no goal on this page.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (10)
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Prescription only
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Oral
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
RNAWiki has not recorded a supervision or regulatory status for this substance.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
No register row and no identity class settled the question.
No source is stored against this line.
What is in the pack
Sold as tablet, tablet, coated, given by the oral route.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take∅Nothing found in the sources checked
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
∅Nothing found in the sources checked
No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.
The sources listed were searched and held nothing. That is not the same as nothing existing.
Reports sent to a regulator
These are reports people sent to a regulator. They do not show the medicine caused the reaction.
Nobody counted how many people took the medicine and reported nothing.
The same event can be reported more than once, and many reports are incomplete.
News coverage, lawsuits and new warnings change how often people report.
A count is not a rate and not a risk.
Osilodrostat appears in spontaneous reports to regulators. Across the 3 most-reported reaction terms, 20 reaction mentions were counted. One report can name several reactions.
The recorded terms (3)
adrenal insufficiency — 14 reaction mentions
covid-19 — 4 reaction mentions
cortisol increased — 2 reaction mentions
open-targets-adr · recorded 2026-06-24 · a recorded source, not a stored snapshot
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear◇Read from sources, not yet reviewed
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
The form that was studied
Oral
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
Nothing further is recorded about which forms are sold.
No source is stored against this line.
What is recorded as being sold
9 products list this as an active ingredient in the United States drug directory. 9 of them contain it and nothing else.
FDA National Drug Code directory · 63069-109 · read 2026-08-29
They are sold as powder, tablet, coated and tablet, film coated, taken oral.
FDA National Drug Code directory · 63069-109 · read 2026-08-29
The regulator's established pharmacologic class for it is cortisol synthesis inhibitor [epc], cytochrome p450 11b1 inhibitors [moa] and cytochrome p450 1a2 inhibitors [moa].
FDA National Drug Code directory · 63069-109 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-29
Isturisa is oral at 3 DOSAGE FORMS AND STRENGTHS ISTURISA is available as: 1 mg tablets: Pale yellow, unscored, round, biconvex with beveled edge tablet, debossed "1" on one side. 5 mg tablets: Yellow, unscored, round, biconvex with bevele…, recorded as fda label in effect 2025-11-18 in the United States.
US prescribing information · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · read 2026-08-30
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
RNAWiki could not confidently classify this substance, so no self-experiment plan is offered.
Questions worth asking
Which of the trials of Osilodrostat studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of Osilodrostat are recorded. Results from one form may not transfer to another.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
What happened in people◇Read from sources, not yet reviewed
Check any of this yourself
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Y6581YAW9V
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2286275
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1 approved application covers products containing this substance. The earliest was NDA212801, approved 20200306 to RECORDATI RARE.
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Recorded evidence blocks (11)
Q1
What did Osilodrostat's largest trial (206 people) and its longest (6.2 years) measure?
206 people in Osilodrostat's largest registered study, 6.2 years in its longest registered window, measuring Percentage of Primary Efficacy Responder at Week 34 by Randomized Treatment and Strata. ClinicalTrials.gov · 2026-09-01
4 phase2, 4 phase4, 3 na or unstated, 2 phase1, 2 phase3; NCT03708900; 2027-07-21; no ageing endpoint recorded. Last human test completed 2023, NCT03606408.
Interpretation These counts include studies where Osilodrostat was a comparison treatment, a background treatment or an exposure in an observational study, and the longest window may be a planned end date. Trial roles have not yet been classified for this record.
Show the evidence
phase2
4
phase4
4
na or unstated
3
phase1
2
phase3
2
Last recorded human testNCT03606408
2023-11-16
recorded 2026-09-01 · last checked 2026-09-04
Q2
From rat to human: where has Osilodrostat shown mechanism-only?
Osilodrostat's half-life is 4 hours — which schedules were studied?
4 hours, the half-life Osilodrostat's label states. openfda-label · f3a5ec24-63c3-4d83-b1c0-6c550fbe7ae2 · 2026-08-30
bioavailability 20% %.
Show the evidence
half life
4 hours hours; Elimination The elimination half-life of osilodrostat is approximately 4 hours.
tmax
12.3 Pharmacokinetics Absorption Osilodrostat is absorbed with a time of maximum observed concentration (T max ) of approximately 1 hour.
bioavailability
20% %; Race/Ethnicity The relative bioavailability in Asian patients is approximately 20% higher compared to that of non-Asian, along with higher T max and C max , compared to other ethnicities.
metabolism
In an absorption, distribution, metabolism, and excretion study, the majority of the radioactivity dose of osilodrostat is eliminated in the urine (mean: 90.6% of administered dose) with only a minor amount eliminated in the feces (1.58% of dose).
recorded 2026-08-30 · last checked 2026-09-04
Q5
Could one person measure Osilodrostat's effect on adverse events?
Adverse events: measured in Osilodrostat's trials.
Interpretation adverse events is the recorded endpoint.
Show the evidence
biomarkers
adverse events; 2026-09-01
adrenal insufficiency; 2026-09-01
fasting glucose hba1c; 2026-09-01
bone mineral density; 2026-09-01
body composition; 2026-09-01
comparison of the efficacy of metyrapone and osilodristat; 2026-09-01
half life
2026-09-04; halfLife; hours; 4 hours; 2026-08-30
human trials at or under30
6
smallest human trial
9; NCT02468193; PHASE2; the smallest recorded human enrolment at or below 30; the registry states no direction of its result; COMPLETED
Q6
Which of adrenal insufficiency, adverse events and body composition did Osilodrostat's trials measure?
adrenal insufficiency, adverse events and body composition lead 6 outcome terms across Osilodrostat's trials. ClinicalTrials.gov · 2026-09-01
Interpretation bone mineral density, body composition and comparison of the efficacy of metyrapone and osilodristat follow.
Show the evidence
adverse events
1
adrenal insufficiency
1
fasting glucose hba1c
1
bone mineral density
1
body composition
1
comparison of the efficacy of metyrapone and osilodristat
1
recorded 2026-09-01 · last checked 2026-09-04
Q7
Which of Osilodrostat's 8 ongoing trials reports first?
Core Study: Evaluate the pharmacokinetics (PK) of osilodrostat using Pharmacokinetic parameters of osilodrostat up to Week 12 in children and adolescents 2 to less than 18 years…; Incidence of osilodrostat-related adverse events and serious adverse events; latest 2029-03
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Trial
NCT03708900
"Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome"; n 12; "Core Study: Evaluate the pharmacokinetics (PK) of osilodrostat using Pharmacokinetic parameters of osilodrostat up to Week 12 in children and adolescents 2 to less than 18 years of age with Cushing's Syndrome"; 2027-07-21
NCT05382156
"Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome"; n 206; "Incidence of osilodrostat-related adverse events and serious adverse events"; 2027-08
NCT06430528
"A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement"; n 12; "Percent of participants who experience an adrenal insufficiency event during Phase 1 (titration phase)"; 2027-08
NCT07104812
"Impact of 1 mg Osilodrostat Therapy on Mild Autonomous Cortisol Secretion (MACS)"; n 15; "Adverse Events"; 2027-01
NCT07247058
"Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)"; n 10; "Change in fasting glucose, HbA1c"; 2029-03
NCT07247162
"Osilodrostat in Patients With Hypertension Caused by Hypercortisolaemia Due to Cushing's Syndrome"; n 63; "To evaluate the efficacy of osilodrostat on the proportion of participants with normalisation of urinary-free cortisol (UFC)"; 2028-07
2 further recorded trials
NCT07268222
"Metyrapone Versus Osilodrostat in Patients With Metabolic Autonomous Cortisol Secretion (MACS)"; n 150; "Comparison of the efficacy of metyrapone and osilodrostat"; 2027-11-25
NCT07603466
"Combination Osilodrostat and Cabergoline in Cushing's Disease"; n 50; "Changes in serum cortisol"; 2028-08
recorded 2026-09-01 · last checked 2026-09-04
Q8
Which 2 trials of Osilodrostat posted no result?
Posted no result
2 of 2 completed trials
Registrations
NCT02399202 and NCT02372084
Completion dates
oldest 2016-03-21; newest 2016-05-19
Show the evidence
Trial
NCT02399202
2016-03-21
NCT02372084
2016-05-19
Q9
At the median, Osilodrostat's trials enrolled 50 people — anything larger?
Median enrolment
50
Largest enrolment
206
Registered trials counted
15
Q10
What do 20 spontaneous reports say about Osilodrostat — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Osilodrostat appears in spontaneous reports to regulators. Across the 3 most-reported reaction terms, 20 reaction mentions were counted: adrenal insufficiency 14; covid-19 4; cortisol increased 2. open-targets-adr · CHEMBL3099695 · 2026-06-24
Show the evidence
adrenal insufficiency
14
covid-19
4
cortisol increased
2
recorded 2026-06-24 · last checked 2026-09-04
Q11
Osilodrostat and CYP2D6, CYP3A4 and CYP1A2: shared by which compounds?
CYP2D6, CYP3A4 and CYP1A2 appear in Osilodrostat's recorded interaction sentences, 7 in all. openfda-label+europepmc · 2026-08-30
Interpretation pharmacokinetics
Show the evidence
CYP1A2pharmacokinetics
Drug Interaction In a healthy volunteer study (N = 20) using a single dose of osilodrostat (50 mg) and a probe drugs cocktail, osilodrostat showed inhibition potential on CYP1A2, CYP2C19, CYP2D6, and CYP3A4/5 isozymes with 2.5-, 1.9-, 1.5- and 1.5-fold increase in caffeine, omeprazole, dextromethorphan, and midazolam exposure, respectively [see Drug Interactions (7.1) , (7.2) ] .
CYP2B6pharmacokinetics
Metabolism Multiple CYP enzymes (i.e., CYP3A4, CYP2B6, and CYP2D6) and UDP-glucuronosyltransferases contribute to osilodrostat metabolism and no single enzyme contributes greater than 25% to the total clearance.
CYP2C19pharmacokinetics
Drug Interaction In a healthy volunteer study (N = 20) using a single dose of osilodrostat (50 mg) and a probe drugs cocktail, osilodrostat showed inhibition potential on CYP1A2, CYP2C19, CYP2D6, and CYP3A4/5 isozymes with 2.5-, 1.9-, 1.5- and 1.5-fold increase in caffeine, omeprazole, dextromethorphan, and midazolam exposure, respectively [see Drug Interactions (7.1) , (7.2) ] .
CYP2D6
pharmacokinetics
Metabolism Multiple CYP enzymes (i.e., CYP3A4, CYP2B6, and CYP2D6) and UDP-glucuronosyltransferases contribute to osilodrostat metabolism and no single enzyme contributes greater than 25% to the total clearance.
pharmacokinetics
Drug Interaction In a healthy volunteer study (N = 20) using a single dose of osilodrostat (50 mg) and a probe drugs cocktail, osilodrostat showed inhibition potential on CYP1A2, CYP2C19, CYP2D6, and CYP3A4/5 isozymes with 2.5-, 1.9-, 1.5- and 1.5-fold increase in caffeine, omeprazole, dextromethorphan, and midazolam exposure, respectively [see Drug Interactions (7.1) , (7.2) ] .
CYP3A4
pharmacokinetics
Metabolism Multiple CYP enzymes (i.e., CYP3A4, CYP2B6, and CYP2D6) and UDP-glucuronosyltransferases contribute to osilodrostat metabolism and no single enzyme contributes greater than 25% to the total clearance.
pharmacokinetics
Drug Interaction In a healthy volunteer study (N = 20) using a single dose of osilodrostat (50 mg) and a probe drugs cocktail, osilodrostat showed inhibition potential on CYP1A2, CYP2C19, CYP2D6, and CYP3A4/5 isozymes with 2.5-, 1.9-, 1.5- and 1.5-fold increase in caffeine, omeprazole, dextromethorphan, and midazolam exposure, respectively [see Drug Interactions (7.1) , (7.2) ] .
recorded 2026-08-30 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Europe PMC — metadata only, under the recorded legal gate · Open Targets 26.06 — CC0 · derived from this page's own recorded fields; no external licence · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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