Lancet Neurology, a prestigious journal in its domain, publishes in its last issue, an article entitled "Pitolisant versus placebo and modafinil in patients with narcolepsy: a double-blind randomised trial" authored by Y. Dauvilliers and others from the HARMONY I study group which proposes a novel treatment for this orphan disease. It is accompanied by a Commentary entitled "A need for new treatments in narcolepsy".
Narcolepsy is a rare disabling disorder mainly characterised by Excessive Daytime Sleepiness (EDS) and cataplexy, a sudden loss of muscular tone following various emotions. Present treatments comprise psychostimulants, like modafinil or amphetamine-like compounds, to fight EDS and sodium oxybate or antidepressants against cataplexy attacks. However these treatments are not fully satisfactory in terms of efficacy, tolerance or administration.
Pitolisant is the first representant of a new class of drugs to be introduced in the clinics, the histamine H3-receptor antagonists; it was designed in 3 European academic laboratories and developed by Bioprojet, a small independent French pharmaceutical company.
The HARMONY I trial comprised 95 narcoleptic patients suffering from severe EDS, among which those treated during 2 months with pitolisant, once a day orally, showed significant improvement over placebo on self-reported and laboratory-assessed EDS as well as on frequency of cataplexy attacks, i.e. the two major symptoms of narcolepsy. Whereas the pitolisant results on EDS did not differ significantly from those of twice-a-day modafinil, a currently used comparator, it was well tolerated compared with modafinil and did not show any manifestation of addiction potential associated with the use of typical psychostimulants of the amphetamine type.
If these findings are substantiated in further, currently ongoing, studies, pitolisant could offer a new treatment option for patients with narcolepsy.