Orzeyful receives global approvals for narcolepsy type 1 treatment

Orzeyful (oveporexton), a first-in-class oral orexin receptor 2 (OX2R) agonist, has received regulatory approvals in the United States, Japan, and China for the treatment of narcolepsy type 1.

Unlike traditional therapies that focus on managing individual symptoms, Orzeyful targets the underlying cause of narcolepsy type 1: the deficiency of orexin (hypocretin), a neuropeptide essential for stabilizing wakefulness, sleep, and muscle tone. The drug works by selectively stimulating the OX2R receptor to restore signaling in the brain.

In clinical Phase 3 trials, the medication demonstrated significant improvements in excessive daytime sleepiness and cataplexy compared to a placebo. In China, the National Medical Products Administration approved the therapy for adults and adolescents aged 16 and older. In Japan, the Ministry of Health, Labour and Welfare approved its use for adults. The FDA also granted approval for adult use.