CAMBRIDGE, Mass. — Takeda said the U.S. Food and Drug Administration has accepted its New Drug Application for oveporexton and granted the therapy Priority Review, marking a key regulatory milestone for a potential first-in-class treatment for narcolepsy type 1.
Oveporexton, also known as TAK-861, is an investigational oral orexin receptor 2–selective agonist designed to address the underlying orexin deficiency that causes narcolepsy type 1 by restoring orexin signaling. The FDA has set a Prescription Drug User Fee Act target action date in the third quarter of this calendar year. Takeda said it remains on track to potentially bring the first approved orexin agonist therapy to people living with the condition.
Narcolepsy type 1 is a chronic and rare neurological disorder caused by a loss of orexin, a neuropeptide critical for regulating wakefulness. The disease is characterized by excessive daytime sleepiness and cataplexy, or sudden loss of muscle tone, and can significantly affect physical functioning, cognition, work, education, and social life. Takeda said many patients continue to experience symptoms despite currently available treatments.
“The FDA’s acceptance of our NDA is a milestone for people living with narcolepsy type 1,” said Andy Plump, M.D., Ph.D., president of R&D 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 application is supported by data from the global Phase 3 FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) studies. According to Takeda, the trials demonstrated statistically significant and clinically meaningful improvements across a broad range of symptoms, including wakefulness, excessive daytime sleepiness, cataplexy, attention, quality of life, and daily functioning, with results approaching near-normal ranges on several measures.
Oveporexton was generally well tolerated across clinical studies, with the most commonly reported adverse events including insomnia, urinary urgency, and urinary frequency.
The therapy has previously received Breakthrough Therapy designation from the FDA for the treatment of excessive daytime sleepiness in narcolepsy type 1, as well as similar recognition from China’s National Medical Products Administration. In Japan, oveporexton has been granted Sakigake designation by the Ministry of Health, Labour and Welfare.
Takeda said the NDA filing is not expected to have a significant impact on its full-year consolidated forecast for the fiscal year ending March 31, 2026. (Source: IANS)
