Takeda’s ORZEYFUL Approved in China for Narcolepsy Type 1

Takeda China announced that the National Medical Products Administration of China has approved the use of ORZEYFUL (oveporexton) for the treatment of narcolepsy type 1 (NT1) in adolescents aged 16 years and older and adults. Oveporexton is the first oral orexin receptor 2 (OX2R)-selective agonist approved in China and is the only approved medicine to treat NT1. In addition to securing approval in China, the new drug application is currently under priority review in the United States, where it received Breakthrough Therapy designation, and under review in Japan, where it received Sakigake designation. Additional regulatory submissions are planned throughout the year.

“As the first and only approved medicine to treat the broad spectrum of narcolepsy type 1, oveporexton has the potential to fundamentally change how the disease is managed. We sincerely appreciate the support from China’s regulatory authorities in enabling an accelerated review process, helping to address a significant unmet medical need here in China,” said Yan Liu, General Manager of Takeda China. “This milestone also marks an important step for Takeda China as we expand our local presence into neuroscience with the aim to advance care for disorders that have a profound impact on people and society.”

Narcolepsy is a chronic, rare neurological disease affecting an estimated 700,000 people in China with NT1 accounting for approximately 75–80 percent of cases. NT1 is driven by orexin deficiency, making it harder to regulate sleep and wake and stabilize transitions between them. As a result, people experience a range of daytime and nighttime symptoms including excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), disrupted nighttime sleep, sleep paralysis, hallucinations and may also experience cognitive symptoms.

NT1 typically develops between the ages of 10 and 20. The persistent, 24-hour nature of the disease can severely impact many aspects of a person’s life, including work, education and social interactions. Furthermore, NT1 may also be associated with an increased risk of traffic accidents, as well as a higher burden of anxiety, depression and related mental health conditions.^1,2 Despite the substantial disease impact, limited awareness and the complexity of symptoms can contribute to misdiagnoses and an average diagnostic delay of more than 10 years.

“For years, treatment options for narcolepsy type 1 have largely relied on symptomatic therapies to manage individual symptoms,” said Professor Han Fang, Director of the Sleep Medicine Center at Peking University People’s Hospital and Director of the Peking University Sleep Research Center. “The approval of oveporexton represents a meaningful paradigm shift in narcolepsy type 1 treatment, signaling a transition from partial symptom management to a mechanism-driven approach that can help address the many symptoms evaluated in clinical studies and may support improvements in daily functioning and quality of life.”

“Oveporexton was internally discovered and developed at Takeda and is the first approved orexin agonist in China to treat narcolepsy type 1,” said Lin Wang, R&D head of global regions and head of R&D China and the Asia-Pacific region at Takeda. “Neuroscience is a strategic focus area for Takeda, and we are committed to translating innovative science into meaningful treatment options for people living with sleep-wake disorders and other areas of unmet need where orexin plays a role.”

The approval is based on the comprehensive clinical program including the global Phase 3 FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) studies that showed statistically significant improvements versus placebo on prespecified endpoints across the NT1 symptoms assessed. This included improvements in excessive daytime sleepiness, cataplexy and other secondary measures evaluated in the studies. Oveporexton demonstrated a safety profile consistent with previous clinical trial data to date. The most common adverse events were insomnia, urinary urgency and urinary frequency. Learn more about the Phase 3 data results here.

Source: Takeda