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Patients with type 1 narcolepsy (NT1) typically experience excessive daytime sleepiness (EDS) and cataplexy (a sudden, brief loss of muscle tone triggered by an emotional event) and may have hypnagogic hallucinations, hypnopompic hallucinations, or sleep paralysis. A loss of orexin-producing neurons in the hypothalamus leads to NT1. Oveporexton, an oral orexin receptor-2–selective agonist that targets this underlying mechanism, has now been studied in two industry-sponsored, phase 3 trials.
Nearly 275 adults with NT1 and at least four cataplexy episodes per week were randomized to receive twice daily (1 mg or 2 mg) oveporexton or placebo for 12 weeks. Participants were off other narcolepsy treatments for at least 2 weeks. Numerous con…