NARCOLEPSY IS A CHRONIC SLEEP DISORDER that can have debilitating consequences.

The disorder typically begins in childhood or adolescence, with mild initial symptoms that can worsen with time.^4,5 Narcolepsy is increasingly recognized as an autoimmune disorder,¹ whereby the immune system reacts to an external trigger (eg, viral) in such a way that it attacks the brain cells (ie, hypocretin neurons) that synthesize a neuropeptide (ie, hypocretin) that regulates wakefulness and sleep.⁴ However, the exact cause remains unknown; genetic risk factors or environmental triggers may precede the loss of hypocretin neurons and subsequent hormone imbalances characteristic of the disorder. Because of heterogeneity in the development and severity of symptoms, many individuals do not receive narcolepsy diagnosis until years after onset.

Jazz Pharmaceuticals, a leading global biopharmaceutical company, recorded an astonishing $1.8 billion in revenue this year from the sales of Gamma Hydroxybutyrate (GHB).

Xyrem® and Xywav® are the trade names of the Food and Drug Administration (FDA)-approved prescription medication versions of GHB approved as a treatment for narcolepsy and manufactured by Jazz.

Narcolepsy is a long-term neurological disorder that impairs the brain’s ability to regulate sleep-wake cycles.

It can cause severe disruptions to a person’s life, manifesting symptoms like excessive daytime sleepiness, sleep paralysis, hallucinations, and in some cases, cataplexy — a sudden loss of muscle control and strength.

Mechanism of Action

• GHB is categorized as a central nervous system (CNS) depressant. Its effects include euphoria, drowsiness, decreased anxiety, confusion and memory impairment.
• GHB can also produce both visual hallucinations and paradoxically, excited and aggressive behavior. GHB greatly increases CNS depressant effects of alcohol and other depressants.
• Its mechanism of action, while not entirely understood, is believed to function by stimulating the GABA receptor in the brain, a primary inhibitory neurotransmitter that slows down brain activity. This action results in a deep sleep state, which helps people with narcolepsy get the restorative sleep they require but often struggle to achieve.

Dosing

The dosing of Xyrem® (GHB) is a carefully managed process. It is usually given in two nightly doses, starting with a lower dose to assess patient tolerance, and then gradually increased to therapeutic levels as tolerated. The total nightly dose ranges from 6 to 9 grams, divided into two doses. The first dose is typically taken at bedtime, and the second dose is taken 2.5 to 4 hours later.