What is Wakix - Pitolisant used for and what is it used for?
Wakix is a medicine used in adults to treat narcolepsy. Narcolepsy is a long-term sleep disorder that compromises the brain's ability to regulate the normal sleep-wake cycle. This causes symptoms such as an irrepressible need to sleep, even in moments and in inappropriate places, and disturbed nocturnal sleep. Some patients also have episodes of severe muscle weakness (cataplexy), sometimes up to collapse. Wakix is used in patients with or without cataplexy.
Wakix contains the active ingredient pitolisant. Because the number of patients with narcolepsy is low, the disease is considered "rare" and Wakix was designated an "orphan medicine" (a medicine used in rare diseases) on 10 July 2007.
How is Wakix - Pitolisant used?
Wakix can only be obtained with a prescription and treatment should be started by a doctor with experience in sleep disorders.
Wakix is available as tablets (4.5 and 18 mg). During the first week of treatment the recommended dose is 9 mg a day, taken in the morning during breakfast. During the second week of treatment, the daily dose can be increased to 18 mg or reduced to 4.5 mg.
During the third week, the dose can be further increased to the maximum dose of 36 mg per day. Wakix should always be used at the lowest effective dose.
In patients with moderate hepatic impairment or with kidney problems, the maximum daily dose should not exceed 18 mg.
For more information, see the package leaflet.
How does Wakix - Pitolisant work?
Wakix contains the active ingredient pitolisant, which binds to receptors in the brain called "H3 histamine receptors". This increases the activity of certain brain cells called "histaminergic neurons", which are important for keeping the body awake.
What benefit has Wakix - Pitolisant shown during the studies?
Wakix was analyzed in 2 main studies involving a total of 261 adults with narcolepsy, in most cases associated with cataplexy. The studies compared Wakix with a placebo (a dummy treatment). The main efficacy parameter was the feeling of sleepiness of patients during the day, assessed by Epworth Sleepiness Scale (ESS). This is the standard scale used in patients with narcolepsy, which has a score ranging from 0 to 24.
The first study showed that Wakix was more effective than placebo in reducing daytime sleepiness: after 8 weeks of treatment, patients taking Wakix had an average reduction on the ESS scale 3 points higher than those taking the placebo. The results of this study also showed a decrease in the number of cataplexy attacks. In the second study, however, there were no differences between Wakix and placebo regarding the reduction of sleepiness or cataplexy.
In an objective test of sleepiness called the Maintenance of Wakefulness Test (MWT), the combined results of the two studies showed a significant improvement in the ability to maintain wakefulness with Wakix compared to placebo.
In another study of 105 patients with narcolepsy and cataplexy, Wakix was more effective than placebo in reducing the weekly number of cataplexy attacks: the number of cataplexy attacks per week had increased from about 9 to about 3 in the patients they took Wakix, while it remained around 7 a week in patients taking placebo.
What is the risk associated with Wakix - Pitolisant?
The most common side effects of Wakix (which may affect up to 1 in 10 people) are insomnia (difficulty sleeping), headache, nausea (feeling sick), anxiety, irritability, dizziness, depression, tremor, trouble sleeping, tiredness, vomiting, dizziness (sensation of rotation of the surrounding environment) and dyspepsia (heartburn). Serious but rare side effects are abnormal weight loss and miscarriage. For the full list of all side effects reported with Wakix, see the package leaflet.
Wakix should not be used in patients with severe impairment of liver function and breast-feeding women. For the full list of limitations, see the package leaflet.
Why has Wakix - Pitolisant been approved?
The overall data available shows that Wakix has a positive effect on the two main symptoms of narcolepsy, excessive daytime sleepiness and cataplexy. Furthermore, Wakix has a different mechanism of action than currently available treatments and therefore represents an alternative therapeutic option. The safety profile of Wakix is considered acceptable, without any other major safety concerns having been identified.
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Wakix's benefits are greater than its risks and recommended that it be approved for use in the EU.
What measures are being taken to ensure the safe and effective use of Wakix - Pitolisant?
A risk management plan has been developed to ensure that Wakix is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Wakix, including the appropriate precautions to be followed by healthcare professionals and patients.
In addition, the company that markets Wakix will conduct an observational study to gather information on the safety of the medicine used in medical practice.
Further information is available in the summary of the risk management plan
More information on Wakix - Pitolisant
For the full version of EPAR and the summary of the Wakix risk management plan, see the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Wakix therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Wakix Committee for Orphan Medicinal Products is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
