FDA Approves First Drug to Treat a Cause of Narcolepsy

But now there’s a drug that addresses one of the biological mechanisms involved in narcolepsy. On Aug. 5, the U.S. Food and Drug Administration approved Orzeyful, or oveporexton, which targets the orexin pathway that orchestrates the brain’s sleeping and wake cycles. Without orexin, the brain loses the ability to control when the body sleeps and when it’s awake. Orzeyful is especially effective for disrupted sleep in narcolepsy type 1, which can be riskier because people lose muscle control and can injure themselves when they do. The drug activates the orexin pathway that is dysfunctional in people with the condition, restoring the brain’s ability to remain alert during the day, and is approved for people with narcolepsy type 1. Based on two studies submitted by Takeda, which developed the drug, involving more than 270 people with the condition, people taking the drug, which comes in pill form, twice a day, were better able to stay awake during the day than those taking a placebo. They also had fewer episodes of cataplexy, or losing muscle control, as well as other symptoms such as irregular nighttime sleep and hallucinations.