Overview
- The U.S. Food and Drug Administration has approved Orzeyful (oveporexton), a twice-daily oral treatment for adults with narcolepsy type 1.
- Orzeyful works as a first-in-class orexin receptor 2 (OX2R) agonist that directly targets the orexin deficiency that causes the disorder.
- The approval was based on two clinical studies in which patients taking Orzeyful showed greater ability to stay awake during the day than those given placebo.
- In trials the most common side effects reported included insomnia, increased urination frequency and urgency, and increased saliva production, and no new liver-toxicity signal was reported for this formulation.
- Takeda estimates narcolepsy type 1 affects about one in 2,000 people in the U.S., and the drug’s approval could shift treatment from symptom control toward addressing the underlying loss of orexin neurons and prompt further study of orexin agonists in related sleep and neurologic conditions.