Narcolepsy is characterized by drowsiness, often during the daytime. This is a symptom that greatly affects the lives of those with narcolepsy, and for a long time no FDA approved treatments existed that were not also listed as controlled substances. Now, thanks to Harmony Biosciences, there is a new treatment available for excessive daytime sleepiness (EDS). Two clinical studies have been conducted on this treatment, WAKIX, and they have been published in SLEEP. These studies were meant to evaluate the safety of the drug along with the potential for abuse.
Narcolepsy is a neurological disorder that stops the brain from regulating one’s sleep-wake cycle. It is characterized by instantly falling asleep for a few minutes or longer, depending on the severity of one’s symptoms. Some people experience hallucinations while asleep or directly after waking up. People often have cataplexy, which is the sudden loss of muscle control, along with narcolepsy, but this is not the case for everybody. When people have both disorders it is known as type one narcolepsy. Cataplexy is triggered by a strong emotion, such as fear or anger, and it manifests as uncontrollable muscle weakness or paralysis. Those who have cataplexy without narcolepsy are often misdiagnosed, as doctors think it is a seizure disorder. Depending on severity, cataplexy can be slight eyelid drooping or the inability to remain standing. One is usually awake during a cataplectic attack but are unable to move. As cataplexy is often related to narcolepsy, people fall asleep after an attack.
The most common cause of an attack is laughter. The destruction of a neurotransmitter hypocretin, which regulates wakefulness, is the root of cataplexy. The cause of narcolepsy type two, which is narcolepsy without cataplexy, is unknown. It is believed that genetics play a part in the disorder, but it is not often passed down from parent to child. Symptoms of type two narcolepsy include sleep paralysis, hallucinations, and changes in rapid eye movement (REM) sleep. In terms of treatment, there are drugs available to treat cataplexy. Xyrem, which was approved in 2002 by the FDA, is used to treat narcolepsy itself, but due to its high potential for abuse it is tightly regulated. Besides drugs, people with narcolepsy often take regularly scheduled naps and do not drink alcohol or caffeine before bed.
Studies on WAKIX
WAKIX was developed by Harmony Biosciences and it is the only FDA approved drug to treat narcolepsy that is not classified as a controlled substance. It was given orphan drug designation in 2010, and in 2016 received approval from the European Medicines Agency. It is approved for the treatment of excessive daytime sleepiness caused by narcolepsy for adults. Two clinical studies, which were published by SLEEP, evaluated the safety, efficacy, and potential for abuse. These studies examined contradictions, precautions, adverse effects, drug interactions, and the use in specific populations. In terms of side effects, the study found that insomnia, nausea, and anxiety were all possibilities, but all occurred in less than 10% of those studied. The studies concluded that there is long-term safety and efficacy of the drug, and that it does not have a high potential for abuse.
About Harmony Biosciences
Harmony Biosciences, located in Plymouth Meeting, Pennsylvania, mainly deals with providing treatments for rare, orphan diseases. They focus on diseases that affect the central nervous system. Narcolepsy is a disorder that they are dedicated to developing treatments for, and they also focus on advancing the understanding of the disorder. Education for those living with narcolepsy is an important factor for Harmony Biosciences.
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