Novel Therapeutic Opportunity in Narcolepsy
PARIS, October 9, 2013 /PRNewswire/ –
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
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
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.
For more details see below:
Jean-Charles Schwartz firstname.lastname@example.org +33-(0)299280440