FDA Approves Oveporexton for Narcolepsy Type 1, First Drug to Treat Full Range of NT1 Symptoms

The US Food and Drug Administration has approved Takeda Pharmaceuticals’ Orzeyful (oveporexton) for treatment of narcolepsy type 1 in adults.¹ The medication targets orexin signaling loss, addressing the full range of narcolepsy symptoms, and is the first approved orexin agonist for narcolepsy type 1.

“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.¹ “This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole,” she added.

Approval was based on 2 successful randomized, double-blind, placebo-controlled trials.^2,3 Across both 12-week studies, 273 adults with narcolepsy type 1 were enrolled. Compared with placebo, patients taking Orzeyful showed improvements in ability to stay awake during daytime and decreased daytime sleepiness. Patients treated also showed a significant reduction in cataplexy episodes and improvement across narcolepsy symptoms including sleep paralysis, hallucinations, and disrupted nighttime sleep. Most common adverse effects from these trials were insomnia, increased urinary frequency, urgency to urinate, and increased saliva production. Rates of participants stopping treatment due to adverse effects were low, and over 95% of participants completing the initial study enrolled in the ongoing long-term extension study.

Orzeyful is an oral 2 mg tablet intended to be taken twice daily. The drug is an orexin receptor 2 selective agonist, selectively stimulating the orexin receptor 2 to restore signaling and address the underlying orexin deficiency in narcolepsy type 1. With this receptor activation, the medication promotes wakefulness and reduces abnormal rapid eye movement sleep-like phenomena (including cataplexy) to address daytime and nighttime symptoms. Developers of the medication noted specifically that Orzeyful should not be used at the same time as strong CYP3A inhibitors.

Approval came after previous Breakthrough Therapy Designation and Priority Review from the FDA in February 2026.⁴ Scheduling for Orzeyful has been recommended under the Controlled Substances Act, with a scheduling decision from the Drug Enforcement Administration pending.

References

1. FDA approves first drug to treat the full range of narcolepsy type 1 symptoms. Press release. August 5, 2026. Accessed August 5, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms

2. A study of TAK-861 for treatment of narcolepsy type 1. ClinicalTrials.gov. July 1, 2025. Accessed August 5, 2026. https://clinicaltrials.gov/study/NCT06470828

3. New pivotal study data show Takeda’s oveporexton improved daily function, cognition and nighttime sleep for people with narcolepsy type 1. Press release. June 15, 2026. Accessed August 5, 2026. https://www.takeda.com/newsroom/newsreleases/2026/oveporexton-phase-3-narcolepsy-type-1/

4. Kuntz L. NDA accepted and priority review granted: oveporexton for narcolepsy type 1. Psychiatric Times. February 10, 2026. https://www.psychiatrictimes.com/view/nda-accepted-and-priority-review-granted-oveporexton-for-narcolepsy-type-1