Skip to content

Macitentan

  • Prescription medicine
  • Prescription only
  • Identity checked
  • Sources last checked 2026-09-04

This page shows what was measured, who it was measured in, and what that does not settle.

What Macitentan does in the body

Pulmonary arterial hypertension

From the FDA-approved label: Endothelin (ET)-1 and its receptors (ET A and ET B ) mediate a variety of deleterious effects, such as vasoconstriction, fibrosis, proliferation, hypertrophy, and inflammation. In disease conditions such as PAH, the local ET system is upregulated and is involved in vascular hypertrophy and in organ damage. Macitentan is an endothelin receptor antagonist that inhibits the binding of ET-1 to both ET A and ET B receptors. Macitentan displays high affinity and sustained occupancy of the ET receptors in human pulmonary arterial smooth muscle cells.

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
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

What the registries record it as

  • The substance registry classes this as chemical.

    FDA substance registry · Z9K9Y9WMVL · read 2026-08-29

  • Its recorded molecular formula is C19H20Br2N6O4S, weighing 588.27.

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-30

Where each sentence above came from

No recorded sentence describes the change this makes in a way that stands on its own. 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 105 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 peopleRead 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.

Double-blind Treatment Period: Time to First Clinical Events Committee (CEC)-adjudicated Morbidity or Mortality (M/M) Events

The study did not show it

Who was studied
NCT04273945
How many people
935
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
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

Time to First Confirmed Morbidity or Mortality Event up to the End of Treatment (Kaplan-Meier Estimate of Patients Without a Morbidity or Mortality Event)

The study did not show it

Who was studied
NCT00660179
How many people
742
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
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 Treatment Emergent Adverse Events (TEAEs) up to 28 Days After Study Treatment Discontinuation

The study did not show it

Who was studied
NCT00667823
How many people
550
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

The change of 6MWD at month 12 from baseline

The study did not show it

Who was studied
NCT06968962
How many people
376
Study design
Not applicable
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

Incidence Rate of New Digital Ulcers (DUs) up to Week 16

The study did not show it

Who was studied
NCT01474109
How many people
289
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

Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT)

The study did not show it

Who was studied
NCT01841762
How many people
284
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 peopleRead 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.

  1. Living longer, or avoiding a major event Evidence recorded. Death, a heart attack, a stroke, a hospital stay.1 registered measure of this kind. No reviewed result.
  2. What a body can do day to day No evidence recorded. Walking, dressing, breathing, recovering.No registered study measures this.
  3. Measured performance No evidence recorded. How much was lifted, how far was run, how fast.No registered study measures this.
  4. Symptoms and quality of life No evidence recorded. Pain, tiredness, mood, sleep, as the person rated it.No registered study measures this.
  5. A number that stands in for health Evidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.1 registered measure of this kind.
  6. A step measured inside a person Evidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
  7. Animals Evidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.Stored records in Mouse, Rat. A result in animals says what to test next. It does not say what happens in people.
  8. Cells in a dish No evidence recorded. Cells or chemistry on a bench, far from a whole body.No cell or bench record is stored.
  9. A guess from software No 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 takeRead 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.

  • maximum plasma concentration of riociguat

Meaningful

Things that change how a life goes, not only a number.

  • teaes leading to death

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 (5)
  • treatment emergent adverse events
  • treatment emergent serious adverse events
  • pulmonary vascular resistance
  • pulse rate over time
  • frequency of treatment emergent adverse events

These are harms, study-process measures, or names the rules did not recognise. They are shown rather than dropped.

What is missing or unclearRead 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.

  • Macitentan tablets are an endothelin receptor antagonist (ERA) indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) in adults to reduce the risks of disease progression and hospitalization for PAH ( 1.1 ).

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 macitentan in pediatric patients have not been established for the treatment of PAH.”

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-30

  • On older people, the label states: “Of the total number of subjects in the clinical study of macitentan for PAH, 14% were 65 and over.”

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-30

  • On people who are pregnant, the label states: “Risk Summary Based on data from animal reproduction studies, macitentan may cause embryo-fetal toxicity, including birth defects and fetal death, when administered to a pregnant female and is contraindicated during pregnancy.”

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-30

  • On people who are breastfeeding, the label states: “8.3 Females and Males of Reproductive Potential Based on data from animal reproductive toxicity studies, macitentan can cause fetal harm, including birth defects and fetal death, when administered to a pregnant patient and is contraindicated during pregnancy [see Contraindications (4.1) , and Use in Specific Populations (8.1) ].”

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · 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 takeRead 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 takeRead 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

Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.

Quoted from a source

Where this came from

Copied from a source RNAWiki stored, with the source named.

Suppression classes recorded: S3, S6.

No source is stored against this line.

What is in the pack

Sold as tablet, tablet, film 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 takeNothing 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.

Macitentan appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 23040 reaction mentions were counted. One report can name several reactions.

The recorded terms (10)
  • dyspnoea — 4852 reaction mentions
  • headache — 2916 reaction mentions
  • nausea — 2221 reaction mentions
  • diarrhoea — 2179 reaction mentions
  • pneumonia — 2139 reaction mentions
  • fatigue — 1999 reaction mentions
  • hospitalisation — 1862 reaction mentions
  • fluid retention — 1653 reaction mentions
  • malaise — 1643 reaction mentions
  • dizziness — 1576 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 unclearRead 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

  • 30 products list this as an active ingredient in the United States drug directory. 26 of them contain it and nothing else.

    FDA National Drug Code directory · 11722-058 · read 2026-08-29

  • They are sold as powder, tablet and tablet, film coated, taken oral.

    FDA National Drug Code directory · 11722-058 · read 2026-08-29

  • The regulator's established pharmacologic class for it is endothelin receptor antagonist [epc] and endothelin receptor antagonists [moa].

    FDA National Drug Code directory · 11722-058 · read 2026-08-29

  • 15 published labels name it as an active ingredient. 14 of them describe this substance alone, which is where its own label text on this page comes from.

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-29

  • Those labels are classed as human prescription drug.

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · read 2026-08-29

  • Macitentan is oral at 3 DOSAGE FORMS AND STRENGTHS Tablets: 10 mg, white to off white, round shaped, biconvex, film coated tablets debossed with ‘L1’ on one side and plain on the other side. • Tablet: 10 mg, recorded as fda label in effect 2025-11-10 in the United States.

    US prescribing information · 93f0ab4a-4756-4915-a000-ed65b26cd61c · 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 takeRead 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.

Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.

Questions worth asking

  • Which of the trials of Macitentan 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 unclearRead 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 Macitentan 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 peopleRead from sources, not yet reviewed

Check any of this yourself

Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.

How many documents were read

  • 14 documents were read for this substance.

    RNAWiki source record

  • 14 of them state the same proteinBinding, and they agree.

    RNAWiki source record

Where else this substance is registered

FDA substance identifier (UNII)
Z9K9Y9WMVL
RxNorm concept
1442137

Checks this page had to pass

  • Passed

    Identity resolved

    no open identity hold

  • Passed

    No unresolved merge across substance families

    no quarantine open

  • Passed

    Every public sentence names a source

    The opening statement carries the origin: Written into the record, not signed off.

  • Not passed

    Trial roles classified for highlighted evidence

    No registered study is classified as testing this substance.

  • Passed

    No internal keys in reader text

    enforced by the copy-contract test over the rendered page

  • Passed

    Safety mode resolved

    Suppression classes recorded: S3, S6.

  • Passed

    Canonical metadata present

    slug and display name present

What is missing or unclearRead from sources, not yet reviewed

How this medicine reached us

Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.

What the approval register records

  • 15 approved applications cover products containing this substance. The earliest was NDA204410, approved 20131018 to ACTELION.

    Drugs@FDA application register · NDA204410 · read 2026-08-29

  • Marketing status on the register: none (tentative approval) and prescription.

    Drugs@FDA application register · NDA204410 · read 2026-08-29

  • The earliest marketing start date recorded for a listed product is 20131018.

    FDA National Drug Code directory · 11722-058 · read 2026-08-29

What is missing or unclearRead from sources, not yet reviewed

What to learn next

Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.

This order is fixed in code and does not count clicks or time on the page.

What is not here

7 questions this page could not answer

These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.

  • What was measured, goal by goal — found nothing in the sources checked.
  • The path through the body — found nothing in the sources checked.
  • How long anything takes — found nothing in the sources checked.
  • What it may clash with — found nothing in the sources checked.
  • Other ways to the same goal — found nothing in the sources checked.
  • Claims that go past the evidence — found nothing in the sources checked.
  • What changed on this page — found nothing in the sources checked.

The record as stored

The full record, for auditing

Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.

Recorded evidence blocks (12)

What did Macitentan's largest trial (2686 people) and its longest (12 years) measure?


2686 people in Macitentan's largest registered study, 12 years in its longest registered window, measuring Time to First Confirmed Morbidity or Mortality Event up to the End of Treatment (Kaplan-Meier Estimate of Patients Without a Morbidity or Mortality Event). ClinicalTrials.gov · 2026-09-01

25 phase3, 15 phase1, 11 phase2, 5 phase4, 1 na or unstated; NCT00667823; 2020-12-07; no ageing endpoint recorded. Last human test completed 2025, NCT02932410.

Interpretation These counts include studies where Macitentan 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
  • phase3
    25
  • phase1
    15
  • phase2
    11
  • phase4
    5
  • na or unstated
    1
  • Last recorded human test NCT02932410
    2025-11-27

recorded 2026-09-01 · last checked 2026-09-04

From mouse to human: where has Macitentan shown lifespan?


mouse: mechanism-only, rat: mechanism-only and human: lifespan (55): the rungs where Macitentan has a recorded finding. Europe PMC and ClinicalTrials.gov · organism ladder · 2026-09-01

Interpretation Time to First Confirmed Morbidity or Mortality Event up to the End of Treatment (Kaplan-Meier Estimate of Patients Without a Morbidity or Mortality Event) — the recorded outcome words.

Yeast C. elegans Drosophila Mouse mechanism-onlyRat mechanism-onlyDog Non-human primate Human lifespan
Show the evidence
  • mouse
    mechanism-only
  • rat
    mechanism-only
  • human NCT00660179
    lifespan; Time to First Confirmed Morbidity or Mortality Event up to the End of Treatment (Kaplan-Meier Estimate of Patients Without a Morbidity or Mortality Event); 55

recorded 2026-09-01 · last checked 2026-09-04

15 of Macitentan's trials stopped: safety, futility/efficacy, accrual/recruitment, funding/business, sponsor decision unspecified, other?


safety (1), futility/efficacy (2), accrual/recruitment (4), funding/business (1), sponsor decision unspecified (1) and other (6): Macitentan's stop wording, clustered. ClinicalTrials.gov · 2026-09-01

"Due to Protocol AC-055B201 (MUSIC) not meeting it's primary end point"; 15 of 55 registered studies

Show the evidence

Trial

  • NCT01346930
    withdrawn; "Due to Protocol AC-055B201 (MUSIC) not meeting it's primary end point"
  • NCT01474122
    terminated; "company decision"
  • NCT01499251
    terminated; "Results did not clearly support continuing development in recurrent GBM"
  • NCT01739400
    terminated; "Primary endpoint of parent study AC-055-305/MAESTRO (NCT01743001) not met."
  • NCT01847014
    terminated; "Protocol-defined criterion of macitentan availability achieved."
  • NCT02081690
    terminated; "Slow recruitment"
9 further recorded trials
  • NCT02254954
    terminated; "Sponsor decision due to low recruitment"
  • NCT02651272
    terminated; "Unable to enroll IRB approved sample of participants."
  • NCT03714815
    terminated; "the absence of a positive trend in all efficacy parameters indicates that macitentan 10 mg does not have a beneficial effect in patients with HFpEF and PVD."
  • NCT03726398
    withdrawn; "Did not identify eligible candidates"
  • NCT03775421
    terminated; "As The RUBATO DB study (NCT03153137) did not show any benefit of treatment with macitentan in Fontan-palliated participants, the sponsor has decided to terminate the RUBATO OL study (NCT03775421). No new safety observations were made."
  • NCT03809650
    terminated; "Due to a change in the development strategy"
  • NCT04271475
    terminated; "The Sponsor decided to stop the study for futility based on a recommendation by the IDMC following a pre-planned interim analysis"
  • NCT05731492
    withdrawn; "Sponsor Decision"
  • NCT05946811
    withdrawn; "Study was cancelled by funding agency before starting enrollment."

recorded 2026-09-01 · last checked 2026-09-04

Human studies of Macitentan used macitentan 3mg — over how long?


studies of Macitentan used the recorded amount. ClinicalTrials.gov · 2026-09-01

13 recorded entries; human; tablet; also "macitentan 3mg", "macitentan 10mg", "Macitentan 3 mg"

Show the evidence

human

  • NCT01474109
    macitentan 3mg
  • NCT01474109
    macitentan 10mg
  • NCT01474122
    Macitentan 3 mg
  • NCT01474122
    Macitentan 10 mg
  • NCT01739400
    tablet; Macitentan 10 mg tablet, once daily.
  • NCT02050802
    Macitentan 30 mg
7 more recorded rows
  • human NCT02554903
    Macitentan 10mg
  • human NCT03714815
    macitentan 10 mg
  • human NCT03726398
    Opsumit 10 Mg Tablet
  • human NCT04273945
    Macitentan 37.5 mg
  • human NCT04273945
    Macitentan 75 mg
  • human NCT04540744
    FDC of macitentan/tadalafil (10 mg/20 mg)
  • human NCT05236231
    Macitentan 10 mg/Tadalafil 20mg FDC

recorded 2026-09-01 · last checked 2026-09-04

Which of frequency of treatment emergent adverse events, maximum plasma concentration of riociguat and pulmonary vascular resistance did Macitentan's trials measure?


frequency of treatment emergent adverse events, maximum plasma concentration of riociguat and pulmonary vascular resistance lead 7 outcome terms across Macitentan's trials. ClinicalTrials.gov · 2026-09-01

Interpretation maximum plasma concentration of riociguat, teaes leading to death, pulse rate over time and frequency of treatment emergent adverse events follow.

Show the evidence
  • treatment emergent adverse events
    1
  • treatment emergent serious adverse events
    1
  • pulmonary vascular resistance
    1
  • maximum plasma concentration of riociguat
    1
  • teaes leading to death
    1
  • pulse rate over time
    1
1 more recorded row
  • frequency of treatment emergent adverse events
    1

recorded 2026-09-01 · last checked 2026-09-04

Which of Macitentan's 3 ongoing trials reports first?


3 registered trials of Macitentan are open; earliest completion 2026-12-31. ClinicalTrials.gov · 2026-09-01

Double-blind Treatment Period: Time to First Clinical Events Committee (CEC)-adjudicated Morbidity or Mortality (M/M) Events; Change in end-systolic elastance (Ees) divided by arterial elastance (Ea) (Ees/Ea) at three time points: Baseline, Timepoint 1 and Timepoint 2.; latest 2030-09-30

Show the evidence

Trial

  • NCT04273945
    "Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension"; n 935; "Double-blind Treatment Period: Time to First Clinical Events Committee (CEC)-adjudicated Morbidity or Mortality (M/M) Events"; 2029-02-20
  • NCT05140525
    "Effects of Combination Medical Therapy Followed by BPA on Right Ventricular-PA Coupling and Hemodynamics in CTEPH"; n 15; "Change in end-systolic elastance (Ees) divided by arterial elastance (Ea) (Ees/Ea) at three time points: Baseline, Timepoint 1 and Timepoint 2."; 2026-12-31
  • NCT05179876
    "A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option"; n 280; "Frequency of Treatment Emergent Adverse Events (TEAEs)"; 2030-09-30

recorded 2026-09-01 · last checked 2026-09-04

Which running trial of Macitentan could settle lifespan?


NCT04273945 measures Double-blind Treatment Period: Time to First Clinical Events Committee (CEC)-adjudicated Morbidity or Mortality (M/M) Events, reading out 2029-02-20.

1 open trial; n 935; "Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension"

Show the evidence
  • Trial NCT04273945
    "Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension"; n 935; "Double-blind Treatment Period: Time to First Clinical Events Committee (CEC)-adjudicated Morbidity or Mortality (M/M) Events"; 2029-02-20

Which 15 trials of Macitentan posted no result?


Posted no result
15 of 15 completed trials
Registrations
NCT02050802, NCT02476864, NCT03215966, NCT03359291, NCT03389321 and NCT02112487, and 9 more
Completion dates
oldest 2011-11; newest 2023-12-27
Show the evidence

Trial

  • NCT02050802
    2011-11
  • NCT02476864
    2015-12
  • NCT03215966
    2017-09-24
  • NCT03359291
    2017-12-04
  • NCT03389321
    2018-02-06
  • NCT02112487
    2018-09-19
9 further recorded trials
  • NCT02126943
    2020-04-24
  • NCT04235270
    2020-07-17
  • NCT04211272
    2021-04-19
  • NCT04540744
    2021-08-30
  • NCT04963439
    2021-10-05
  • NCT05236231
    2022-05-14
  • NCT05392530
    2022-09-14
  • NCT05433675
    2022-10-03
  • NCT03422328
    2023-12-27

At the median, Macitentan's trials enrolled 75 people — anything larger?


Median enrolment
75
Largest enrolment
2686
Registered trials counted
55

What do 23040 spontaneous reports say about Macitentan — 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.

Macitentan appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 23040 reaction mentions were counted: dyspnoea 4852; headache 2916; nausea 2221; diarrhoea 2179. open-targets-adr · CHEMBL2103873 · 2026-06-24

Show the evidence
  • dyspnoea
    4852
  • headache
    2916
  • nausea
    2221
  • diarrhoea
    2179
  • pneumonia
    2139
  • fatigue
    1999
4 more recorded rows
  • hospitalisation
    1862
  • fluid retention
    1653
  • malaise
    1643
  • dizziness
    1576

recorded 2026-06-24 · last checked 2026-09-04

Macitentan and CYP3A4, CYP2C9 and P-GP: shared by which compounds?


CYP3A4, CYP2C9 and P-GP appear in Macitentan's recorded interaction sentences, 7 in all. openfda-label+europepmc · 2026-08-30

Interpretation pharmacokinetics

Show the evidence
  • CYP2C19 pharmacokinetics
    This reaction is dependent on the cytochrome P450 (CYP) system, mainly CYP3A4 with minor contributions of CYP2C8, CYP2C9, and CYP2C19.
  • CYP2C8 pharmacokinetics
    This reaction is dependent on the cytochrome P450 (CYP) system, mainly CYP3A4 with minor contributions of CYP2C8, CYP2C9, and CYP2C19.

CYP2C9

  • pharmacokinetics
    This reaction is dependent on the cytochrome P450 (CYP) system, mainly CYP3A4 with minor contributions of CYP2C8, CYP2C9, and CYP2C19.
  • pharmacokinetics
    PBPK modeling and simulations based analysis showed that a moderate dual inhibitor of CYP3A4 and CYP2C9 such as fluconazole (400 mg once daily) is predicted to increase macitentan exposure approximately 4-fold without relevant effect on the exposure to its active metabolite [see Drug Interactions (7.3) ] .

CYP3A4

  • pharmacokinetics
    This reaction is dependent on the cytochrome P450 (CYP) system, mainly CYP3A4 with minor contributions of CYP2C8, CYP2C9, and CYP2C19.
  • pharmacokinetics
    Figure 1 Effects of other strong CYP3A4 inhibitors such as ritonavir on macitentan were not studied, but are likely to result in an increase in macitentan exposure at steady state similar to that seen with ketoconazole [see Drug Interactions (7.2) ] .
  • pharmacokinetics
    PBPK modeling and simulations based analysis showed that a moderate dual inhibitor of CYP3A4 and CYP2C9 such as fluconazole (400 mg once daily) is predicted to increase macitentan exposure approximately 4-fold without relevant effect on the exposure to its active metabolite [see Drug Interactions (7.3) ] .

recorded 2026-08-30 · last checked 2026-09-04

Was Macitentan studied with exercise?


exercise is named in Macitentan's label sentences: "The primary end point of RUBATO-DB was not met; macitentan did not improve exercise capacity versus placebo in patients with Fontan palliation." openfda-label+europepmc · 2026-08-30

1 recorded statement; exercise

Show the evidence
  • exercise
    The primary end point of RUBATO-DB was not met; macitentan did not improve exercise capacity versus placebo in patients with Fontan palliation.

recorded 2026-08-30 · last checked 2026-09-04

Where it is registered
Identifiers, relations and other names

The exact record

ChEMBL id
CHEMBL2103873
PubChem CID
16004692
CAS number
441798-33-0
RxCUI
1442132
InChIKey
JGCMEBMXRHSZKX-UHFFFAOYSA-N
Development code
ACT 064992
Trade name
Macitentan component of opsynvi, Opsumit, OPSYNVI COMPONENT MACITENTAN, Macitentan Accord, Macitentan AccordPharma, Yuvanci
Also called
Macitentan [INN], Macitentan [JAN], Macitentan [MI], Macitentan [ORANGE BOOK], Macitentan [USAN], Macitentan [VANDF], Macitentan [WHO-DD], N-(5-(4-BROMOPHENYL)-6-(2-((5-BROMOPYRIMIDIN-2-YL)OXI)ETHOXY)PYRIMIDIN-4-YL)-N'-PROPYLSULFURIC DIAMIDE
Sources (9)

Sources

3 more sources
  • openfda-label 226049ba-9c76-0798-6725-ee027a041820 ·
  • openfda-label+europepmc K1:Z9K9Y9WMVL ·
  • national registers US, EU, CA ·

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

How these records are assembled · Which registers were checked

Index-quality checks
  • identity passed: no open identity hold
  • required summary fields resolved: 5 required field(s) not terminal: Why people use it, Best-supported result, Most important common problem, Biggest unanswered question, Human evidence
  • public claims reviewed: 0 reviewed claim(s); drafts are never rendered
  • source coverage passed: 9 source rows
  • no critical contamination: no quarantine open
  • canonical metadata passed: slug and display name present
  • no raw internal fields: enforced by the copy-contract test over the rendered page

This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.