This page shows what was measured, who it was measured in, and what that does not settle.
What Pitolisant does in the body
Excessive daytime sleepiness (EDS) or cataplexy
From the FDA-approved label: The mechanism of action of pitolisant in excessive daytime sleepiness (EDS) or cataplexy in patients 6 years and older with narcolepsy is unclear. However, its efficacy could be mediated through its activity as an antagonist/inverse agonist at histamine-3 (H3) receptors.
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 · 4BC83L4PIY · read 2026-08-29
Its recorded molecular formula is C17H26ClNO·HCl, weighing 332.31.
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · 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 64 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.
Biomarker
A biomarker is a number from a test that stands in for something about health.
A picture of it, and where the picture fails
A biomarker is like a fuel gauge.
Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.
What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.
A measurable indicator used as a substitute for a clinical outcome of interest.
Placebo
A placebo is a dummy treatment given so the real one can be compared with it.
A picture of it, and where the picture fails
A placebo is like a blank control in an experiment.
Where that stops being true. A blank does nothing. People given a placebo often do get better.
What people get wrong. A placebo effect is read as imaginary. The improvement is measured and real.
An inactive intervention matched in appearance to the test intervention, used to control for non-specific effects.
Comparator
A comparator is whatever the treatment was measured against.
A picture of it, and where the picture fails
It is like the other runner in a race.
Where that stops being true. A race has one winner. A study can show both arms improved.
What people get wrong. Results are read without asking what the other group got. Beating nothing is not beating a treatment.
The control condition against which the experimental intervention is assessed.
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.
Epworth sleepiness scale (ESS)
✗ The study did not show it
Who was studied
NCT02739568
How many people
389
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. Film coated
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
ESS change (Epworth Sleepiness Scale) versus baseline
✗ The study did not show it
Who was studied
NCT01066442
How many people
273
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. Film coated
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
ESS change (Epworth Sleepiness Scale)
✗ The study did not show it
Who was studied
NCT01036139
How many people
268
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. Film coated
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
ESS (Epworth Sleepiness Scale) change from baseline
✗ The study did not show it
Who was studied
NCT01072968
How many people
268
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. Film coated
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 in severity of EDS as measured by the Epworth Sleepiness Scale (ESS)
✗ The study did not show it
Who was studied
NCT07675135
How many people
258
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. Film coated
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 in severity of EDS as measured by the Epworth Sleepiness Scale (ESS)
✗ The study did not show it
Who was studied
NCT07500090
How many people
248
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. Film coated
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 healthNo evidence recorded. Cholesterol, blood sugar, bone density. A stand-in, not the thing itself.No registered study measures this.
■A step measured inside a personEvidence recorded. Something measured in human tissue or human cells.A human record sits on the stored organism ladder.
□AnimalsNo evidence recorded. Mice, rats, dogs. Useful for ideas, not proof about people.No animal record is stored.
□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
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. 20 hours hours
Read from the label, which states: “Elimination After a single dose of 35.6 mg, the median half-life of pitolisant is approximately 20 hours (7.5 to 24.2 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.
WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in patients 6 years of age and older with narcolepsy. WAKIX is a histamine-3 (H3) receptor antagonist/inverse agonist indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy patients 6 years of age and older with narcolepsy ( 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 WAKIX have not been established for the treatment of excessive daytime sleepiness or cataplexy in pediatric patients less than 6 years of age with narcolepsy.”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-30
On older people, the label states: “Limited pharmacokinetic data are available in healthy elderly subjects.”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-30
On people who are pregnant, the label states: “Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to WAKIX during pregnancy.”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary The transfer of pitolisant into breastmilk is low based on data from a lactation study.”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-30
On people with reduced liver function, the label states: “WAKIX is contraindicated in patients with severe hepatic impairment (Child-Pugh Class C) as it has not been studied in this population.”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-30
On people with reduced kidney function, the label states: “The pharmacokinetics of WAKIX in patients with end-stage renal disease (ESRD) (eGFR of <15 mL/minute/1.73 m 2 ) is unknown [see Clinical Pharmacology ( 12.3 )] .”
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · 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
Film coated
✎ 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, film coated, given by the film coated, 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.
Pitolisant appears in spontaneous reports to regulators. Across the 9 most-reported reaction terms, 31 reaction mentions were counted. One report can name several reactions.
The recorded terms (9)
depression — 7 reaction mentions
anxiety — 5 reaction mentions
initial insomnia — 4 reaction mentions
nightmare — 4 reaction mentions
dry mouth — 3 reaction mentions
restless legs syndrome — 3 reaction mentions
feeling jittery — 2 reaction mentions
splenic infarction — 2 reaction mentions
prehypertension — 1 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
Film coated
✎ 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
12 products list this as an active ingredient in the United States drug directory. 12 of them contain it and nothing else.
FDA National Drug Code directory · 46016-9030 · read 2026-08-29
They are sold as powder and tablet, film coated, taken oral.
FDA National Drug Code directory · 46016-9030 · read 2026-08-29
The regulator's established pharmacologic class for it is cytochrome p450 3a4 inducers [moa], histamine h3 receptor antagonists [moa] and histamine h3 receptor inverse agonists [moa].
FDA National Drug Code directory · 46016-9030 · 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 · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · read 2026-08-29
Wakix is oral at 3 DOSAGE FORMS AND STRENGTHS WAKIX 4.45 mg tablets: white, round, biconvex film-coated tablet, marked with “S” on one side and plain on the other side., recorded as fda label in effect 2026-02-13 in the United States.
US prescribing information · 8daa5562-824e-476c-9652-26ceef3d4b0e · 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 Pitolisant 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 Pitolisant 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
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Where else this substance is registered
FDA substance identifier (UNII)
4BC83L4PIY
CAS registry number
362665-56-3
PubChem compound
9948102
ChEMBL
CHEMBL462605
WHO international nonproprietary name list entry
8952
RxNorm concept
2197878
EMA substance identifier
100000161112
DrugBank
DB11642
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
✗ Not passed
Safety mode resolved
No register row and no identity class settled the question.
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read 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
5 approved applications cover products containing this substance. The earliest was NDA211150, approved 20190814 to HARMONY.
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.
Felt, measured, or meaningful — 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)
Q1
What did Pitolisant's largest trial (1329 people) and its longest (9 years) measure?
1329 people in Pitolisant's largest registered study, 9 years in its longest registered window, measuring Mesure of classic pharmacokinetic parameters determined on BF2.649 serum and urine concentration. ClinicalTrials.gov · 2026-09-01
17 phase3, 6 phase1, 6 phase2, 2 na or unstated, 1 phase4; NCT02611687; 2025-06-05; no ageing endpoint recorded. Last human test completed 2026, NCT04257929.
Interpretation These counts include studies where Pitolisant 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
17
phase1
6
phase2
6
na or unstated
2
phase4
1
Last recorded human testNCT04257929
2026-01-21
recorded 2026-09-01 · last checked 2026-09-04
Q2
Pitolisant was tested only in human — what did it show?
4 recorded entries; human; also "BF 2.649 10 mg", "BF 2.649 20 mg", "BF 2.649 40 mg"
Show the evidence
human
NCT00642928
BF 2.649 5 mg
NCT00642928
BF 2.649 10 mg
NCT00642928
BF 2.649 20 mg
NCT00642928
BF 2.649 40 mg
recorded 2026-09-01 · last checked 2026-09-04
Q5
Pitolisant's half-life is 20 hours — which schedules were studied?
20 hours, the half-life Pitolisant's label states. openfda-label · 8daa5562-824e-476c-9652-26ceef3d4b0e · 2026-08-30
Show the evidence
half life
20 hours hours; Elimination After a single dose of 35.6 mg, the median half-life of pitolisant is approximately 20 hours (7.5 to 24.2 hours).
tmax
Absorption The median time to maximum plasma concentration (T max ) of pitolisant is 3.5 hours (2 to 5 hours).
metabolism
Metabolism Pitolisant is primarily metabolized by CYP2D6 and to a lesser extent by CYP3A4; these metabolites are further metabolized or conjugated with glycine or glucuronic acid.
recorded 2026-08-30 · last checked 2026-09-04
Q6
Could one person measure Pitolisant's effect on epworth sleepiness scale scores?
Epworth sleepiness scale scores: measured in Pitolisant's trials.
Interpretation epworth sleepiness scale scores is the recorded endpoint.
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biomarkers
epworth sleepiness scale scores; 2026-09-01
epworth sleepiness scale; 2026-09-01
incidence of treatment emergent adverse event; 2026-09-01
maximum effect on drug liking visual analog scale; 2026-09-01
0; NCT02800083; PHASE2; the smallest recorded human enrolment at or below 30; the registry states no direction of its result; WITHDRAWN
Q7
Which of epworth sleepiness scale, epworth sleepiness scale scores and excessive daytime sleepiness did Pitolisant's trials measure?
epworth sleepiness scale, epworth sleepiness scale scores and excessive daytime sleepiness lead 7 outcome terms across Pitolisant's trials. ClinicalTrials.gov · 2026-09-01
Interpretation maximum effect on drug liking visual analog scale, excessive daytime sleepiness, major congenital malformation and reporting treatment emergent adverse events follow.
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epworth sleepiness scale scores
1
epworth sleepiness scale
1
incidence of treatment emergent adverse event
1
maximum effect on drug liking visual analog scale
1
excessive daytime sleepiness
1
major congenital malformation
1
1 more recorded row
reporting treatment emergent adverse events
1
recorded 2026-09-01 · last checked 2026-09-04
Q8
Which of Pitolisant's 4 ongoing trials reports first?
Major congenital malformation; Social Responsiveness Scale Second Edition (SRS-2) total score; latest 2030-08
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Trial
NCT05536011
"WAKIX® (Pitolisant) Pregnancy Registry"; n 1329; "Major congenital malformation"; 2030-06-30
NCT05953389
"Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents"; n 62; "Social Responsiveness Scale Second Edition (SRS-2) total score"; 2025-11
NCT06366464
"A Study of Pitolisant in Patients With Prader-Willi Syndrome"; n 134; "Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score"; 2028-04
NCT07219485
"A Study of Pitolisant in Participants With Prader-Willi Syndrome"; n 150; "Percentage of participants reporting Treatment-Emergent Adverse Events (TEAEs)"; 2030-08
recorded 2026-09-01 · last checked 2026-09-04
Q9
Which 17 trials of Pitolisant posted no result?
Posted no result
17 of 17 completed trials
Registrations
NCT00642928, NCT01067235, NCT01067222, NCT01620554, NCT01638403 and NCT01036139, and 11 more
Completion dates
oldest 2009-06; newest 2023-07-18
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Trial
NCT00642928
2009-06
NCT01067235
2010-07
NCT01067222
2010-12
NCT01620554
2011-12
NCT01638403
2012-07
NCT01036139
2012-08
11 further recorded trials
NCT01066442
2012-08
NCT01619033
2012-11
NCT01071876
2014-03
NCT01072968
2014-05
NCT01789398
2014-08
NCT01800045
2015-01
NCT02929342
2016-08
NCT01399606
2016-09
NCT03152123
2017-10-23
NCT02739568
2020-04
NCT05223166
2023-07-18
Q10
At the median, Pitolisant's trials enrolled 103 people — anything larger?
Median enrolment
103
Largest enrolment
1329
Registered trials counted
31
Q11
What do 31 spontaneous reports say about Pitolisant — 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.
Pitolisant appears in spontaneous reports to regulators. Across the 9 most-reported reaction terms, 31 reaction mentions were counted: depression 7; anxiety 5; initial insomnia 4; nightmare 4. open-targets-adr · CHEMBL4164059 · 2026-06-24
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depression
7
anxiety
5
initial insomnia
4
nightmare
4
dry mouth
3
restless legs syndrome
3
3 more recorded rows
feeling jittery
2
splenic infarction
2
prehypertension
1
recorded 2026-06-24 · last checked 2026-09-04
Q12
Pitolisant and CYP2D6 and CYP3A4: shared by which compounds?
CYP2D6 and CYP3A4 appear in Pitolisant's recorded interaction sentences, 6 in all. openfda-label+europepmc · 2026-08-30
Metabolism Pitolisant is primarily metabolized by CYP2D6 and to a lesser extent by CYP3A4; these metabolites are further metabolized or conjugated with glycine or glucuronic acid.
The gene encoding CYP2D6 has variants that affect CYP2D6 metabolic function.
clinical_pharmacology
CYP2D6 poor metabolizers are individuals with two nonfunctional alleles (e.g., CYP2D6*5/*5), and as a result have no CYP2D6 enzyme activity.
clinical_pharmacology
Administration of WAKIX 17.8 mg daily for 7 days resulted in 2.4 times higher AUC and 2.1 times higher C max in CYP2D6 poor metabolizers (n=3) compared to CYP2D6 normal metabolizers (n=5). [see Dosage and Administration ( 2.7 ), Use in Genomic Subgroups ( 8.8 )] .
CYP3A4pharmacokinetics
Metabolism Pitolisant is primarily metabolized by CYP2D6 and to a lesser extent by CYP3A4; these metabolites are further metabolized or conjugated with glycine or glucuronic acid.
recorded 2026-08-30 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
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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