The FDA Approves a New Treatment for Narcolepsy

According to a story from PR Newswire, the biopharmaceutical company Harmony Biosciences, LLC recently announced that the US Food and Drug Administration (FDA) has approved the company’s drug pitolisant (marketed as WAKIX®) as a treatment for excessive daytime sleepiness in adults with narcolepsy, a rare disorder of the sleep-wake cycle. Harmony is focused on the development of innovative therapeutics for rare diseases that impact the central nervous system.

About Narcolepsy

Narcolepsy is a neurological disorder that affects sleep. Patients with narcolepsy have a decreased ability to regulate their cycles of sleeping and wakefulness. The exact cause of narcolepsy is not well known. There are a number of risk factors for the condition however, such as family history, exposure to pesticides, or prior brain injury, such as a stroke or tumor. The most well known symptom is excessive sleepiness during the day, often to the extent that a patient may fall asleep suddenly during their regular activities. This can occur even after a full night of sleep; patients are unable to sleep as deeply as an unaffected person. Other symptoms include cataplexy, hallucinations, sleep paralysis, insomnia, and unexpected weight gain. There is no cure for narcolepsy. Treatments for the condition include stimulants such as amphetamines and modafinil. Most patients cannot control their symptoms entirely. To learn more about narcolepsy, click here.

About Pitolisant (WAKIX)

WAKIX is an important new treatment for narcolepsy, partially because it is the first that currently isn’t listed as a controlled substance by the US Drug Enforcement Agency (DEA). It also utilizes a proprietary mechanism of action as a selective antagonist of the histamine 3 receptor. Histamine is a neurotransmitter that promotes wakefulness and the WAKIX mechanism stimulates the release of histamine. 

The approval of this drug follows two clinical trials in which the drug was compared to placebo and an active control. These trials involved a total of 261 patients with narcolepsy. The majority of these patients also had a history of cataplexy, episodes of abrupt, sudden muscle weakness. The results of these trials revealed that WAKIX was able to demonstrate statistically meaningful improvements in excessive daytime sleepiness.


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