Wakix® Becomes First and Only Non-Controlled Drug Approved for Cataplexy Due to Narcolepsy
Last updated: February 2021
The U.S. Food and Drug Administration (FDA) has approved Wakix® (pitolisant) for the treatment of cataplexy in adults with narcolepsy. This makes Wakix the first and only non-controlled, FDA-approved treatment for people with excessive daytime sleepiness (EDS) or cataplexy associated with narcolepsy.1
Wakix is the first drug in its class and is a selective histamine 3 (H3) receptor antagonist/inverse agonist. Wakix helps make and release histamine, which helps people stay awake.1
Wakix was first approved for the treatment of EDS in adults with narcolepsy in August 2019.1
What is narcolepsy?
Narcolepsy is a rare neurological disorder that affects up to 165,000 people in the United States. People with narcolepsy have EDS, or the inability to stay awake and alert during the day.1
Two-thirds of people with narcolepsy have cataplexy. Cataplexy is a sudden and temporary loss of muscle tone. Intense emotions, like excitement or laughter, can trigger cataplexy. Symptoms can be mild and subtle (drooping eyelids) or severe (knee buckling or total body collapse). Cataplexy can significantly affect daily function.1
Evidence for Wakix
The FDA based the expanded approval of Wakix on results from 2 trials, Harmony CTP and Harmony 1. These studies were randomized and controlled clinical trials. Based on these trials, Wakix was approved to treat EDS due to narcolepsy. Wakix was recently analyzed again as a treatment for cataplexy.2
The Harmony CTP study showed that people who took Wakix had a 75 percent lower weekly cataplexy rate. People who took a placebo (inactive medicine) had a 38 percent lower weekly rate of cataplexy. Also, study data showed that over 1 year, there was a 76 percent decrease in cataplexy. Based on study results, Wakix can now also be used to treat cataplexy in adults.1,2
Side effects of the study were similar to the side effects found when Wakix was in clinical trials. The most common side effects were:2
- Trouble sleeping
- Weight gain
- Anxiety, depression, and irritability
- Nausea and vomiting, and irritability
Why is this an important approval?
Wakix is the first and only non-controlled, FDA-approved treatment for people with EDS or cataplexy due to narcolepsy. Controlled substance medications have the potential for abuse or dependence, as determined by the U.S. Drug Enforcement Administration (DEA). Since Wakix is not a controlled substance, there is no potential for abuse or dependence, making it a safer option.1
People with narcolepsy have excessive daytime sleepiness, and up to two-thirds of people with narcolepsy have cataplexy. Cataplexy is very debilitating. It can significantly impact daily function. This new approval offers another treatment option for people living with cataplexy and EDS.1
Things to think about
Wakix prolongs the QT interval (the time it takes for the heart muscles to contract and recover). Before taking Wakix, talk to your doctor if you have QT prolongation, cardiac arrhythmia, or other heart problems or electrolyte abnormalities. The risk of QT prolongation may be higher in people with liver or kidney problems. Do not use Wakix if you have severe liver problems or end-stage renal disease.1
The most common side effects of Wakix are:1
Other less common side effects include:1
- Upper respiratory infection
- Muscle or abdominal pain
- Increased heart rate
- Decreased appetite
- Dry mouth
These are not all the possible side effects of Wakix. Talk to your doctor about what to expect or if you experience any changes that worry you.
Wakix interacts with certain drugs. Some drugs should not be used with Wakix. Tell your doctor about any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.1
Wakix can interfere with hormonal birth control. If you use hormonal birth control, use a backup, non-hormonal form of birth control while taking Wakix and for at least 21 days after you stop taking Wakix. Tell your doctor if you are pregnant or plan to become pregnant.1
Wakix is not right for everyone. Talk to your doctor about the risks and benefits of Wakix, and whether it is right for you.
For more information, read the full prescribing information for Wakix.
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