Pitolisant: A New Narcolepsy Medication

What is Pitolisant?

Pitolisant is a novel treatment for narcolepsy and works as an inverse agonist/antagonist histamininergic agent, with selectivity for the H3 receptor. Animal studies show that it acts as a stimulant.

The European Medicines Agency (EMA) approved the use of pitolisant (Wakix, Bioprojet Pharma) for treatment of narcolepsy with or without cataplexy. The FDA granted it orphan drug status. Such designation provides the pharmaceutical companies with incentives such as lowered fees for scientific advice and access to patients.

Narcolepsy is a sleep disorder in which the brain’s ability to regulate the normal sleep wake cycle is abnormal. Individuals with narcolepsy often develop excessive sleepiness during the daytime, altered night time sleep and have sudden urges to sleep. Some individuals also experience episodes of cataplexy, which has the potential of causing falls and car accidents. Individuals with narcolepsy have a poor quality of life and there are no good treatments.

European researchers say that Pitolisant will add more treatment options for narcolepsy, by promoting wakefulness and alertness.

Pitolisant Clinical Trial Results

In a recent clinical trial on 259 patients, the drug was found to be effective and relatively safe. The 109 patients treated with Pitolisant were observed to have decreased day time sleepiness and were more alert. The most common side effects observed in the trials were insomnia, nausea and headache. Currently more long term studies are taking place to determine its safety.

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7 thoughts on “Pitolisant: A New Narcolepsy Medication

  1. Ruth Reply

    My father (dec.) and daughter were both diagnosed with narcolepsy and cataplexy. Both have/had severe sleep apnea as well. Daughter also diagnosed with Ehler Danlos syndome. These conditions are tragically under diagnosed. Patients are often thought to be “malingerers” by physicians.

    Perhaps many “chronic fatigue syndrome” patients actually have these illnesses.

    With the advent of evolving DNA health testing I am hopeful that many illnesses with be diagnosed early in life so people are not robbed of functionality most of their lives.

  2. Susan Reply

    Please do not refer to narcolepsy as a “very rare” condition. According to NIH “Narcolepsy is not rare, but it is an underrecognized and underdiagnosed condition.”

    Calling it “rare” only makes it more difficult to get diagnosed with this disabling condition, because doctors think that it’s too rare for any of their patients to have it.

    It’s true that getting an actual diagnosis is relatively rare; I’ve always heard that only 1 in 4 people with narcolepsy are ever diagnosed, and that it can take many years to get the diagnosis.

    I started having symptoms when I was a college student, started having cataplexy in my 40s, and was finally diagnosed in my 50s.

  3. Ann Marie Sahr Reply

    Any new information is welcomed. I have been suffering with narcolepsy (diagnosed) since 1970. I actually feel it began in about 1967. U of M diagnosed me after a internist suspected narcolepsy and sent me to a neurologist who tested me and also agreed it was narcolepsy but wanted to confirm. Her results with U of M. Same. Was confirmed but I was sent away and told there wasn’t any treatment. I finally received medication from the neurologist and my only results have been increasingly worse along with severe cataplexies. This has caused falls with severe head injuries including breaking my teeth and again breaking veneers.

    • Hannah Reply

      Hi, Ann Marie!
      I can’t imagine what your journey has been like, going through life with a disease that too little is known about since the 1967. I too struggle with narcolepsy and all the frustrating things that come with it. I was just diagnosed last year, and have only met one other person with narcolepsy. I found forums to be very helpful. There’s a lot of support there, and it’s a good way to stay updated on new information that comes out. It’s always good to be able to talk with people who understand what you’re going through. If you live near a large-ish city, you may have access to a support group and/or conferences as well.
      I wish you the best,


  4. admin Post authorReply

    Letter to ASA:

    Thank you for your wonderful, informative Article about Narcolepsy. A friend of mine recently found out that she has Narcolepsy at age 60+ years of age, having had symptoms consistent with it since childhood that went undiagnosed. She also recently found out from her mother that her father’s condition that was disabling enough to prevent him from driving was Narcolepsy.

    I am writing to you because from what I have learned from your Article and others from The National Institute of Health it seems that Narcolepsy may be continuing to go undiagnosed in many individuals because parents may not be being educated about the possible genetic predisposition to the condition that their children might have when a close family-member has the condition. Also parents and physicians need to be made aware that the symptoms can express more subtly in many individuals, even though the impact on the person’s life can still be profound.

    My friend’s troubling and somewhat disabling symptoms are much more mild on the surface than her father’s symptoms, yet they have had a profound effect on her life.

    It seems that since it is well-known that this is a disabling condition that very often goes undiagnosed in both children and adults, better family education and education of Physicians of patients with “diagnosed” Narcolepsy is very mush needed. Also Physicians/Pediatricians who often ask about the whole Family Health History need to understand that it can be Genetically-linked even if NOT Genetically-caused which might help more children and adults who are struggling with the symptoms of this condition “in the dark” without knowledge, understanding or treatment get the diagnosis and knowledge they need to better cope with the troubling/disabling symptoms they have been experiencing.

  5. William Adams MD Reply

    I am a Sleep Specialist in the United States. Can one obtain pitolisant (Wakix) legally in the United States?

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