The first drug of its kind to treat patients with the rare but long-term sleep disorder narcolepsy is being recommended for use across the European Union (EU).
The European Medicines Agency (EMA) is recommending that pitolisant (Wakix) is granted a marketing authorisation for the treatment of narcolepsy with or without cataplexy — sudden severe muscle weakness or loss of muscle control.
The drug, a first-in-class medicine, is an antagonist of the histamine H3 receptor, leading to an increase in histamine release in the brain, which in turn enhances wakefulness and alertness.
The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) is based on the results of two placebo-controlled studies involving 259 patients, as well as one uncontrolled, open-label study in 102 patients with narcolepsy and one supportive study in 105 patients.
The studies showed that the drug reduced excessive daytime sleepiness in patients with narcolepsy. Two of the studies revealed benefits for patients who also had cataplexy.
The CHMP has asked the drug manufacturer Bioprojet Pharma to carry out a long-term study in order to further investigate the safety of the medicine, which has had orphan drug status since 2007.
The recommendation, made at the CHMP November 2015 meeting, now has to be approved by the European Commission. It will then be up to individual member states to decide whether to make the drug available within their national health system.