FDA Approves Takeda’s Orzeyful to Treat Type 1 Narcolepsy

The U.S. Food and Drug Administration (FDA) has approved Takeda’s Orzeyful (oveporexton) tablets to treat narcolepsy type 1 in adults. The first medicine approved for narcolepsy type 1 as a complete disorder, it addresses the full range of symptoms that define the condition, working by directly targeting the loss of orexin signaling that causes the disease.

Orzeyful is an oral tablet taken twice daily that, rather than masking symptoms with stimulants or sedatives, directly activates the same brain receptor the body’s own orexin would normally stimulate, restoring the missing signal.  

Approval was based on two randomized, double-blind, placebo-controlled 12-week studies that evaluated the effectiveness and safety of Orzeyful in 273 adults with narcolepsy type 1. In both studies, patients who received 2 mg of Orzeyful showed improvements in their ability to stay awake during the day compared with those receiving placebo. Patients also reported substantially less daytime sleepiness, a significant reduction in cataplexy episodes, and meaningful improvement across the full spectrum of narcolepsy symptoms, including sleep paralysis, hallucinations and disrupted nighttime sleep.

The most common side effects of Orzeyful were insomnia, increased urinary frequency, urgency to urinate and increased saliva production. And, the rate of participants stopping treatment due to side effects was low. 

“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 division of psychiatry within the FDA’s Center for Drug Evaluation and Research. “This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole.”