FDA Alert

FDA Approves Orzeyful for Narcolepsy Type 1 in US Adults

Edited by:
Ashton L. Stahl

Key Highlights
 

  • Oveporexton (Orzeyful) is the first FDA-approved treatment targeting narcolepsy type 1 as a complete disorder.
  • Two 12-week studies enrolled 273 adults with narcolepsy type 1.
  • Treatment improved daytime wakefulness and reduced cataplexy and other symptoms.
  • Common adverse events included insomnia and increased urinary frequency.

On August 5, the FDA approved oveporexton (Orzeyful, Takeda Pharmaceuticals America, Inc) to treat narcolepsy type 1 in adults. Oveporexton is the first medication approved to address the full range of symptoms defining the disorder and the first to directly target the loss of orexin signaling that causes the disease. The medication activates the same brain receptor normally stimulated by the body’s orexin, restoring the missing signal.

The approval was based on 2 randomized, double-blind, placebo-controlled studies involving 273 adults with narcolepsy type 1. During the 12-week studies, patients receiving oveporexton 2 mg demonstrated improved daytime wakefulness compared with placebo. Patients also reported substantially less daytime sleepiness, significantly fewer cataplexy episodes, and meaningful improvements in sleep paralysis, hallucinations, disrupted nighttime sleep, and other narcolepsy symptoms.

“For too long, people with narcolepsy type 1 have had to manage a complex, lifelong neuropsychiatric condition with treatments that only address pieces of it,” Tiffany R. Farchione, MD, director of the Division of Psychiatry within the FDA’s Center for Drug Evaluation and Research, said in a press release. “This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole.”

The most common adverse events associated with oveporexton were insomnia, increased urinary frequency, urinary urgency, and increased saliva production. Few participants discontinued treatment because of adverse events. Oveporexton should not be used concurrently with strong CYP3A inhibitors, and its safety and effectiveness have not been established in patients younger than 18 years.

According to the FDA, oveporexton is administered as an oral tablet twice daily. A specific recommended dose and treatment duration were not provided in the release.

References

US Food and Drug Administration. FDA approves first drug to treat the full range of narcolepsy type 1 symptoms. Published August 5, 2026. Accessed August 20, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms