The investigational oral OX2R-selective agonist is designed to restore orexin signaling; the FDA has set a target action date for the third quarter of 2026.
Key takeaways:
- The FDA accepted the NDA and granted priority review for oveporexton (TAK-861) for narcolepsy type 1 treatment.
- A PDUFA goal date has been set for the third quarter of 2026.
- Oveporexton is designed to address the underlying orexin deficiency in NT1 by restoring orexin signaling.
- The filing is supported by data from the phase 3 FirstLight and RadiantLight studies.
- Common adverse events in trials included insomnia, urinary urgency, and urinary frequency.
The US Food and Drug Administration (FDA) has accepted Takeda’s new drug application (NDA) and granted priority review for oveporexton (TAK-861) for the treatment of narcolepsy type 1 (NT1).
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date in the third quarter of 2026. If approved, oveporexton would potentially be the first orexin agonist treatment for patients with NT1.
Oveporexton is an investigational oral orexin receptor 2 (OX2R)-selective agonist. It is designed to address the specific pathophysiology of NT1 by restoring orexin signaling.
“The FDA’s acceptance of our NDA is a milestone for people living with narcolepsy type 1,” says Andy Plump, MD, PhD, president of R&D at Takeda, in a release. “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.”
The NDA submission is supported by data from the FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) global phase 3 studies. According to Takeda, data measuring objective and patient-reported outcomes showed statistically significant improvements in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, and daily life functions.
In clinical studies to date, oveporexton was generally well-tolerated. The most common adverse events reported were insomnia, urinary urgency, and urinary frequency.
Oveporexton previously received Breakthrough Therapy designation from the FDA for the treatment of excessive daytime sleepiness in NT1. It also received Sakigake designation from the Japanese Ministry of Health, Labour and Welfare.
Takeda noted that the NDA filing has no significant impact on the consolidated forecast for the fiscal year ending March 31, 2026. The company is also developing other orexin agonists, including TAK-360 for narcolepsy type 2 and idiopathic hypersomnia.
