FDA approves Orzeyful to treat narcolepsy type 1

The U.S. Food and Drug Administration has approved Orzeyful (oveporexton), a drug designed to address a biological mechanism of narcolepsy type 1. Developed by Takeda, the medication targets the orexin pathway, which is responsible for regulating the brain’s sleep and wake cycles.

In individuals with type 1 narcolepsy, a deficiency in hypocretin—a brain chemical that maintains wakefulness—often leads to excessive daytime sleepiness and cataplexy, a sudden loss of muscle control. Clinical studies involving over 270 participants showed that patients taking Orzeyful twice daily were better able to remain alert during the day and experienced fewer episodes of cataplexy, hallucinations, and irregular nighttime sleep compared to those on a placebo.

The drug is currently under review by the Drug Enforcement Administration (DEA) to assess its potential for addiction. Takeda CEO Julie Kim expects the medication to receive a schedule IV designation, indicating a low potential for abuse.