Nature头条：晚上睡不着，白天醒不了？首款嗜睡症药物获批，打开大脑新疗法大门

TL;DR - The FDA approved Takeda’s Oveporexton (Orzeyful), the first oral therapy designed to address the biological cause of type 1 narcolepsy by replacing deficient orexin signaling. The approval introduces a potentially broader drug strategy for neurological and psychiatric disorders involving orexin pathways.

• Oveporexton crosses the blood-brain barrier and selectively activates orexin-2 receptors, compensating for orexin-producing neuron loss in type 1 narcolepsy.
• Two phase 3 trials involving 273 patients reported improved daytime wakefulness, nighttime sleep, cataplexy, sleep paralysis, and hallucinations versus placebo.
• Unlike existing symptom-focused treatments, the drug targets the disrupted sleep-wake circuitry underlying the disease.
• Researchers are investigating orexin agonists for other conditions linked to appetite, anxiety, and addiction, while noting that narcolepsy mechanisms beyond orexin deficiency remain unresolved.