FDA Approves Orzeyful, First Orexin-Targeting Drug for Narcolepsy Type 1

The U.S. Food and Drug Administration has approved Takeda's oral orexin‑2 receptor agonist oveporexton, to be marketed as Orzeyful, for adults with narcolepsy type 1. It is the first medication to address the underlying loss of orexin signaling rather than merely managing individual symptoms. In two 12‑week Phase III trials (FirstLight and RadiantLight) involving 273 patients, Orzeyful significantly improved wakefulness and reduced cataplexy, sleep paralysis, hallucinations and other symptoms. The drug received breakthrough‑therapy designation and priority review. "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," said Tiffany R. Farchione, MD, director of the FDA’s Division of Psychiatry. "This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole."

Common side effects reported were insomnia, urinary urgency/frequency and increased saliva production. Orzeyful is indicated for adults only and is not approved for patients under 18. Takeda will market the drug in the United States following the FDA’s approval.