Takeda announced that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for oveporexton and granted it Priority Review for the treatment of narcolepsy type 1 (NT1), a rare chronic neurological disorder. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date in the third quarter of this calendar year.

Oveporexton is an investigational oral orexin receptor 2 (OX2R)-selective agonist designed to address the underlying cause of NT1, orexin deficiency, by restoring orexin signalling. If approved, it could become the first orexin agonist therapy available for people living with NT1, potentially transforming the current treatment landscape.

Narcolepsy type 1 is characterised by excessive daytime sleepiness and cataplexy, a sudden loss of muscle tone. The condition can significantly impair daily functioning, affecting work, education and social interactions. Despite available therapies, many patients continue to experience persistent symptoms, highlighting a substantial unmet medical need.

The NDA submission is supported by data from the global phase III FirstLight and RadiantLight trials. The studies demonstrated statistically significant and clinically meaningful improvements across multiple endpoints, including wakefulness, excessive daytime sleepiness, cataplexy, attention, quality of life and daily functioning. Results showed patients achieving near-normal ranges across a broad spectrum of symptoms. Oveporexton was generally well tolerated, with the most commonly reported adverse events being insomnia, urinary urgency and urinary frequency.

Oveporexton has previously received Breakthrough Therapy designation from the US FDA and China’s National Medical Products Administration (NMPA) for excessive daytime sleepiness in NT1, as well as Sakigake designation from Japan’s Ministry of Health, Labour and Welfare.