NDA Accepted and Priority Review Granted: Oveporexton for Narcolepsy Type 1

Takeda today announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) and granted Priority Review for oveporexton (TAK-861) for the treatment of narcolepsy type 1 (NT1). Oveporexton is an investigational oral orexin receptor 2 (OX2R)-selective agonist specifically designed to address orexin deficiency, an underlying issue that causes NT1, by restoring orexin signaling. If successful, this would be the first approved orexin agonist treatment to individuals living with NT1.¹

NT1 is a chronic, rare neurological disease caused by a loss of orexin that is characterized by excessive daytime sleepiness and cataplexy. NT1 results in a spectrum of physical, cognitive, and psychosocial effects that can have a debilitating impact on an individual’s quality of life, disrupting work, education, and social interactions. While therapies to manage NT1 exist, most patients continue to experience symptoms and are forced to cope with the continued impact of NT1.

“The FDA’s acceptance of our NDA is a milestone for people living with narcolepsy type 1,” said Andy Plump, MD, PhD, president of research and development at Takeda. “Considering the high unmet need, this community deserves a new and different treatment approach that aims to address the underlying orexin deficiency that causes NT1 by restoring orexin signaling. We are one step closer to potentially transforming the current treatment paradigm and intend to deliver through our leading work in orexin science.”

The NDA filing is supported by data from the FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) global phase 3 studies. Both studies met all primary and secondary endpoints demonstrating statistically significant improvement across a broad range of NT1 symptoms compared to placebo with P-values of <0.001 across all doses (twice-daily 1mg/twice-daily 2mg) at week 12, according to data presented at the 2025 World Sleep Congress in Singapore.² Specifically, oveporexton demonstrated objective and patient-reported improvements in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, overall quality of life and daily life functions, achieving near normal ranges across the broad range of symptoms investigated. As to safety profile, oveporexton was generally well-tolerated and consistent across clinical studies to date. The most common adverse events were insomnia, urinary urgency, and urinary frequency.

““Our research has shown that the loss of orexin is the cause of narcolepsy type 1, which results in symptoms like excessive daytime sleepiness and cataplexy,” said Emmanuel Mignot, MD, PhD, principal investigator for the FirstLight phase 3 study. “Takeda’s groundbreaking efforts targeting the orexin receptor 2 in clinical studies led to positive phase 3 results for oveporexton, bringing us a major step closer to having the first orexin therapy that addresses the underlying cause of narcolepsy type 1—with the potential of transforming the current treatment paradigm.”

Oveporexton previously received Breakthrough Therapy designation for the treatment of excessive daytime sleepiness in NT1 from the FDA and the Center for Drug Evaluation of China’s National Medical Products Administration. Oveporexton has also received Sakigake designation from the Japanese Ministry of Health, Labour and Welfare.

The FDA has set a Prescription Drug User Fee Act goal date in the third quarter of 2026. Stay tuned for further updates.

References

1. U.S. Food and Drug Administration accepts new drug application and grants priority review for Takeda’s oveporexton (TAK-861) as a potential first-in-class therapy for narcolepsy type 1. News release. February 10, 2026. Accessed February 10, 2026. https://www.takeda.com/newsroom/newsreleases/2026/fda-accepts-nda-priority-review-oveporexton-narcolepsy-type-1/

2. Takeda presents orexin data from landmark oveporexton (TAK-861) phase 3 program in narcolepsy type 1 at World Sleep 2025. News release. September 8 2025. Accessed February 10, 2026. https://www.takeda.com/newsroom/newsreleases/2025/takeda-orexin-data-oveporexton-phase-3-narcolepsy-world-sleep-2025/