The FDA has approved Wakix to treat cataplexy in children ages 6 and older with narcolepsy.
Narcolepsy affects about 170,000 Americans.
It is often caused by the loss of a brain chemical called hypocretin (also known as orexin), a neuropeptide that regulates and stabilizes sleep‑wake cycles. People with narcolepsy experience overwhelming daytime sleepiness, which makes it difficult for them to stay awake and alert during the day, along with sudden episodes of muscle weakness, brief sleep paralysis, dream-like hallucinations, or vivid dreams while falling asleep or waking up. The condition usually begins in adolescence or young adulthood and affects both men and women equally. Cataplexy can be frightening, socially disruptive, and sometimes dangerous. Unlike many traditional medications for narcolepsy, Wakix is not a stimulant. Instead, it works by selectively blocking the histamine‑3 (H₃) receptor in the brain. Although the exact mechanism is not fully understood, Wakix may help increase the levels of histamine, a neurotransmitter that promotes wakefulness and reduces cataplexy episodes. First approved in 2019, Wakix is now the first and only medicine that is not scheduled as a controlled substance (non-scheduled) — meaning, it does not carry the same abuse or dependence risk as many stimulant-based narcolepsy drugs — to be approved for treating narcolepsy with or without cataplexy in both adults and children 6 or older.
Why Was It Approved?
The FDA’s approval was based on data from a clinical study that showed that Wakix is safe and effective in reducing the number of cataplexy episodes in children with narcolepsy. In this clinical study involving 110 children ages 6-17 with narcolepsy, 86% of the children had a history of cataplexy. The children were randomly selected to receive either Wakix or a placebo once daily, with doses gradually increasing from 4.45 milligrams to 17.8 milligrams for those under 40 kilograms or to 35.6 milligrams for those weighing 40 kilograms or more. Among the 95 children with cataplexy, the average number of weekly cataplexy attacks was significantly reduced to 2.2 with Wakix vs 5.6 with placebo, with a rate ratio of 0.39, showing that Wakix is effective in reducing the number of cataplexy episodes in children. Common side effects were generally manageable and included headaches and insomnia (trouble sleeping). Also, the overall side effect profile of Wakix in this clinical study involving children was consistent with that observed in clinical studies involving adults.
What Do I Need to Know?
Wakix is available as tablets and is taken by mouth once daily in the morning upon wakening. Your health care provider will decide the right dose for you or your child and may gradually increase it over several weeks based on your or your child’s weight and age and how you or your child responds to the medicine. Before starting Wakix, tell your health care provider if you or your child has any liver or kidney problems, heart conditions, or a history of allergic reactions to pitolisant. If you or your child have heart- or kidney-related issues, your health care provider will monitor you or your child during the treatment to detect any heart-related complications. Be sure to tell your health care provider about all the medicines, including over-the-counter products, herbal remedies, and vitamin supplements, that you take as Wakix may interact with them, and this may require dose adjustments to prevent side effects. If you or your child is taking Wakix, always inform your health care provider before starting any new medicine.
