Pitolisant, sold under the brand name Wakix among others, is a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

for the treatment of

excessive daytime sleepiness Excessive daytime sleepiness (EDS) is characterized by persistent sleepiness and often a general lack of energy, even during the day after apparently adequate or even prolonged nighttime sleep. EDS can be considered as a broad condition encompass ...

in adults with

narcolepsy Narcolepsy is a long-term neurological disorder that involves a decreased ability to regulate sleep–wake cycles. Symptoms often include periods of excessive daytime sleepiness and brief involuntary sleep episodes. About 70% of those affec ...

. It is a histamine 3 (H₃) receptor antagonist/

inverse agonist In pharmacology, an inverse agonist is a drug that binds to the same receptor as an agonist but induces a pharmacological response opposite to that of the agonist. A neutral antagonist has no activity in the absence of an agonist or inverse agon ...

. It represents the first commercially available medication in its class. Pitolisant enhances the activity of histaminergic

neurons A neuron, neurone, or nerve cell is an electrically excitable cell that communicates with other cells via specialized connections called synapses. The neuron is the main component of nervous tissue in all animals except sponges and placozoa. N ...

in the brain that function to improve a person's wakefulness. The most common side effects include difficulty sleeping, nausea, and feeling worried. The U.S.

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

(FDA) considers it to be a

first-in-class medication A first-in-class medication is a pharmaceutical that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medicat ...

Medical uses

Pitolisant (Wakix) is

used Used may refer to: Common meanings *Used good, goods of any type that have been used before or pre-owned *Used to, English auxiliary verb Places *Used, Huesca, a village in Huesca, Aragon, Spain *Used, Zaragoza, a town in Zaragoza, Aragon, Spain ...

in adults for the treatment of excessive daytime sleepiness and narcolepsy. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Narcolepsy is a sleep problem that is characterized by an irresistible urge to sleep and disturbed nighttime sleep, while cataplexy refers to attacks of severe muscle weakness that cause a person to collapse. Pitolisant (Ozawade) is indicated to improve wakefulness and reduce excessive daytime sleepiness in adults with

obstructive sleep apnea Obstructive sleep apnea (OSA) is the most common sleep-related breathing disorder and is characterized by recurrent episodes of complete or partial obstruction of the upper airway leading to reduced or absent breathing during sleep. These episod ...

Side effects

The most common side effects include insomnia (difficulty sleeping), headache, nausea (feeling sick), anxiety, irritability, dizziness, depression, tremor, sleep disorders, tiredness, vomiting, vertigo (a spinning sensation) and dyspepsia (heartburn). Serious but rare side effects are abnormal loss of weight and spontaneous abortion.

History

Pitolisant was developed by Jean-Charles Schwartz, Walter Schunack, and colleagues after the former discovered the H₃ receptor. It was the first H₃ receptor inverse agonist to be tested in humans or introduced for clinical use. It is marketed in the European Union by Bioprojet Pharma. It was approved for medical use in the European Union in March 2016. The FDA approved pitolisant for excessive daytime sleepiness in participants with narcolepsy based primarily on evidence from two trials (Trial 1/NCT01067222, Trial 2/NCT01638403). An additional trial (Trial 3/NCT01800045), in which participants with a different type of narcolepsy were exposed to the same dose of pitolisant, was used to add data for evaluation of side effects. The trials were conducted in Europe and South America. The two primary trials enrolled adults with narcolepsy and excessive daytime sleepiness. Participants received pitolisant, placebo, or an approved drug for narcolepsy for eight weeks. For participants receiving pitolisant, the dose could be increased during the first three weeks but had to remain the same for the next five weeks. Neither the participants nor the healthcare providers knew which treatment was being given during the trial. The benefit of pitolisant was evaluated by comparing changes in daytime sleepiness during the trial between pitolisant- and placebo-treated participants. To measure the daytime sleepiness, the investigators used a scale called the Epworth Sleepiness Scale (ESS). The ESS asks participants to rate the likelihood that they would fall asleep while doing eight daily activities (such as sitting and reading or watching television). Participants rate each item from zero (would never doze) to three (high chance of dozing). Pitolisant was approved by the U.S.

(FDA) in August 2019. It was granted

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

designation for the treatment of narcolepsy,

fast track The fast track is an informal English term meaning "the quickest and most direct route to achievement of a goal, as in competing for professional advancement". By definition, it implies that a less direct, slower route also exists. Fast track or F ...

designation for the treatment of

and

cataplexy Cataplexy is a sudden and transient episode of muscle weakness accompanied by full conscious awareness, typically triggered by emotions such as laughing, crying, or terror. Cataplexy affects approximately 70% of people who have narcolepsy, and is ...

in people with narcolepsy, and

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "br ...

designation for the treatment of cataplexy in people with narcolepsy.

References

External links

* * * * Chloroarenes CYP2D6 inhibitors Ethers H3 receptor antagonists 1-Piperidinyl compounds Stimulants Orphan drugs {{Nervous-system-drug-stub