First drug to directly target orexin signaling loss that causes disorder
Narcolepsy type 1 is a rare, lifelong neuropsychiatric sleep disorder caused by the loss of brain cells that produce orexin, a chemical messenger that controls wakefulness, sleep and muscle tone, the FDA said. The condition causes symptoms including excessive daytime sleepiness, cataplexy — sudden muscle weakness caused by strong emotions — sleep paralysis, disrupted nighttime sleep, and hallucinations at the edge of sleep or waking.
Tiffany Farchione, director of the division of psychiatry in the FDA’s Center for Drug Evaluation and Research, said people with narcolepsy type 1 have long had to manage the condition with treatments that only address pieces of it. She said Orzeyful is the first medicine that impacts the underlying biology of the disease, treating the condition as a whole rather than targeting individual symptoms.
Orzeyful works by directly activating the same brain receptor that naturally produced orexin would stimulate, the FDA said. The drug is taken as an oral tablet twice daily.
The approval was granted to Takeda Pharmaceuticals America.