US-based biopharmaceutical firm Harmony Biosciences has secured the US Food and Drug Administration’s (FDA) breakthrough therapy and fast track designations for its drug candidate, pitolisant.
The breakthrough therapy status has been granted to treat cataplexy in people living with narcolepsy, while the fast track designation is to treat excessive daytime sleepiness and cataplexy, which are sudden episodes of muscle weakness, in narcolepsy patients.
Pitolisant is an investigational product being developed as a selective histamine H3-receptor antagonist to boost histaminergic neuron activity in the brain.
This enhanced activity is expected to improve the wakefulness and prevent cataplexy attacks.
Harmony Biosciences chief medical officer Jeffrey Dayno said: “We are very pleased that the FDA has granted pitolisant breakthrough therapy and fast track designations and believe it reflects the Agency’s interest in this investigational product to potentially address an important unmet medical need for patients with narcolepsy.
“We look forward to working with the FDA throughout the submission and review of a new drug application (NDA) for this first-in-class molecule with a novel mechanism of action to treat adult patients with narcolepsy with or without cataplexy.”
Pitolisant has been developed and marketed in Europe by Bioprojet after gaining the European Medicines Agency approval in 2016. Following the latest FDA decision, Harmony is planning for a rolling submission to potentially obtain US regulatory approval next year.
Currently, Harmony is enrolling patients for the Pitolisant Expanded Access Clinical Evaluation (PEACE) programme in order to treat adults with excessive daytime sleepiness due to narcolepsy with or without cataplexy in the US.