Harmony Biosciences Details Long-Term Safety, Efficacy of Narcolepsy Drug

Harmony Biosciences earlier this month presented positive long-term data on its investigational drug pitolisant, which is in development for the treatment of excessive daytime sleepiness and cataplexy (sudden muscle weakening) in adults with narcolepsy. Pitolisant is a potent and selective modulator of the histamine 3 receptor.

The data came from 48 narcolepsy patients who, as part of the Harmony III safety and efficacy trial, took 40 mg pitolisant once daily for up to five years; 32 of these patients remained enrolled for the full period.

The study found that the participants showed continual sleep improvements over the course of five years. Daytime sleepiness, as measured by the Epworth Sleepiness Scale, improved from 16.8 at baseline to 13.4 after one year and 10.6 after five years.

Following one year of treatment, the participants also saw reduced cataplexy (76 percent decline), drowsiness-related hallucinations (54 percent decline), and sleep paralysis (62 percent decline). Harmony noted that there were limited data from patient sleep diaries at the five-year mark to assess these symptoms.

The safety and tolerability profile of pitolisant was consistent with that reported in previous short-term clinical trials. ■