A wakefulness-promoting medicine

Pitolisant

A wakefulness-promoting medicine for narcolepsy, with or without cataplexy, that works through histamine.

What is Pitolisant?

Pitolisant is a medicine for narcolepsy, used with or without cataplexy (sudden muscle weakness triggered by emotion). It works through the brain chemical histamine to increase wakefulness and can also help reduce cataplexy. It is started by a specialist and taken once in the morning. It can affect the heart's rhythm (a change called QT prolongation) and, importantly, it reduces the reliability of hormonal contraception, so additional or alternative methods are needed. Headache, sleep problems and nausea are common.

Education and reference only. This is a plain-language guide to Pitolisant — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.

Brands: Wakix
Pitolisant (Wakefulness (histamine H3 antagonist)) — Meds Global Health reference card with 2D molecular structure
Pitolisant — Wakefulness (histamine H3 antagonist). The image shows the active ingredient's 2D molecular structure.

What it is

Pitolisant is a wakefulness-promoting medicine used to treat narcolepsy, both in people who also have cataplexy and those who do not. Cataplexy is a sudden, brief loss of muscle strength brought on by strong emotion. Unlike older wakefulness medicines, pitolisant works through the brain's histamine system. It is a specialist-initiated tablet, usually taken once in the morning, and the dose is built up gradually when starting.

How it works

Pitolisant acts on histamine in the brain, a chemical messenger involved in keeping us awake. By boosting histamine signalling, it increases wakefulness during the day and can also reduce the frequency of cataplexy attacks in people with narcolepsy. It does not cure narcolepsy but helps control its main symptoms. Because it influences brain wakefulness, it is taken in the morning and built up slowly to find the right level.

Company & origin

Originated / developed by: Bioprojet.

A wakefulness-promoting medicine used in the UK for narcolepsy, with or without cataplexy.

Practical use

How to take Pitolisant

General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.

  • Take it by mouth once in the morning, as your specialist directs; the dose is usually increased gradually at first.
  • Use extra or alternative contraception if you could become pregnant, as pitolisant makes hormonal contraception less reliable.
  • Tell your prescriber about all your other medicines, as some can add to its effect on heart rhythm.
  • Do not stop the medicine suddenly without advice; your specialist will guide any changes.
  • Report palpitations, fainting, chest pain or new mood changes promptly.

Weighing it up

Advantages & disadvantages of Pitolisant

Advantages

  • Treats narcolepsy with or without cataplexy, helping both daytime sleepiness and cataplexy attacks.
  • Works through histamine, offering a different option from stimulant-type wakefulness medicines.
  • Taken once a day in the morning, which is convenient.

Disadvantages

  • Commonly causes headache, sleep difficulty, nausea and anxiety.
  • Can affect the heart's rhythm (QT prolongation), needing care with other medicines.
  • Reduces the reliability of hormonal contraception, so additional methods are needed.

Practical use

Good to know

Pitolisant is started and supervised by a specialist and is usually built up gradually. Common side effects include headache, difficulty sleeping, nausea and anxiety. An important safety point is that pitolisant can affect the heart's electrical rhythm (QT prolongation), so it is used with care alongside other medicines that do the same, and your prescriber may check this. Like the modafinil-type medicines, pitolisant reduces the reliability of hormonal contraception, so you need additional or alternative methods during treatment and for a while afterwards. Do not stop suddenly without advice, and report palpitations, fainting, chest pain or new mood changes such as anxiety or low mood.

Who should not take it / use with caution

  • People with certain heart-rhythm problems, including a tendency to QT prolongation, should not usually take it.
  • It is used with caution in severe liver problems and may be unsuitable for some.
  • People relying only on hormonal contraception need additional cover; it is generally avoided in pregnancy unless advised.

Monitoring

  • Reviewing how well daytime sleepiness and any cataplexy are controlled.
  • Checking the heart's rhythm where appropriate, especially with other QT-affecting medicines.
  • Watching for mood changes and ensuring reliable contraception is in place.

Side effects

  • Headache, difficulty sleeping, nausea and anxiety are among the more common effects.
  • Dizziness, indigestion or an irritable mood in some people.
  • Rarely, changes in heart rhythm (QT prolongation) or significant mood changes, which need prompt review.

Key interactions

  • Reduces the reliability of hormonal contraception such as the pill, patch and implant, so extra methods are needed.
  • Caution with other medicines that can affect heart rhythm (QT prolongation).
  • Some antidepressants and antihistamines, and medicines that change how it is broken down, can interact, so tell your prescriber.

Available as: Tablets taken by mouth.

Answers

Pitolisant: frequently asked questions

What is pitolisant used for?

It is used to treat narcolepsy, with or without cataplexy, increasing daytime wakefulness and helping to reduce cataplexy attacks.

How does it work?

It acts on histamine in the brain, a chemical messenger that helps keep us awake, which is a different approach from stimulant-type medicines.

Does it affect my contraception?

Yes. Pitolisant makes hormonal contraception less reliable, so you should use additional or alternative methods during treatment and for a while afterwards.

Can it affect my heart?

It can affect the heart's electrical rhythm (QT prolongation), so tell your prescriber about your other medicines and report palpitations or fainting.

Should I stop it if I feel better?

Do not stop suddenly without advice; speak to your specialist, who will guide any changes, and report any new mood changes.

Authoritative sources

  • BNF
  • NICE CKS

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