The equivalent of the FDA, the European Medicines Agency (EMA) has approved the use of pitolisant (Wakix, Bioprojet Pharma) for treatment of narcolepsy with or without cataplexy.
Narcolepsy is a very rare sleep disorder where 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.
Pitolisant is an inverse agonist/antagonist histamininergic agent, with selectivity for the H3 receptor. Animal studies show that it acts as a stimulant and may have an application for management of narcolepsy. The drug is not officially approved in the USA but has been granted orphan drug status. Such designation provides the pharmaceutical companies with incentives such as lowered fees for scientific advice and access to patients.
The European researchers say that Pitolisant will add more treatment options for narcolepsy, by promoting wakefulness and alertness.
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.
Edited -Dr. Lin