The US Food and Drug Administration (FDA) has approved Takeda’s Orzeyful (oveporexton), an oral orexin receptor 2 (OX2R) agonist, for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults.
The persistent 24-hour nature of NT1 is driven by orexin deficiency and can severely impact people’s lives.
As a first-in-class orexin treatment, Orzeyful is the only medicine indicated in the US to treat the disease holistically rather than individual symptoms.
NT1 is a rare, chronic neurological disease driven by a loss of orexin and affects an estimated 120,000 people in the US people with NT1 experience excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), cognitive symptoms and disrupted nighttime sleep that can severely impact multiple aspects of daily life, including work, education and social interactions.
The approval is based on a comprehensive clinical programme including the global phase 3 FirstLight and RadiantLight studies that showed oveporexton offers statistically significant improvements across the full range of symptoms of the disease.
These included improvements in excessive daytime sleepiness, cataplexy and health-related quality of life. Oveporexton was generally well-tolerated with a safety profile consistent across clinical studies to date.
