Investigational Narcolepsy Agent Found Effective for Severely Affected Patients with Cataplexy in Phase 3 Study
Results from a phase 3 controlled trial show that pitolisant (Wakix, Bioproject Pharma) may be efficacious in severely affected narcoleptic patients with cataplexy. Pitolisant is a highly-selective histamine H3 receptor inverse agonist/antagonist that is indicated in adults for the treatment of narcolepsy with and without cataplexy. The trial showed that pitolisant, which has previously shown to be active against EDS, was also able to significantly reduce cataplexy when given once-a-day for two months in severely affected patients. The data, published recently in The Lancet Neurology, suggest that pitolisant may represent a first-line treatment of narcolepsy, for which well-tolerated and effective therapeutic options are limited.