New narcolepsy drug reaches Chinese patients on global launch day

Chinese patients gained access to a new class of narcolepsy treatment on Thursday, as a hospital in Beijing issued prescriptions for Takeda's oveporexton on the first day of its global commercial rollout.

The medicine, developed and submitted for regulatory review in China alongside other major markets, was prescribed at Xuanwu Hospital affiliated with Capital Medical University on the first day of its commercial supply simultaneously in China and Japan.

Marketed as Orzeyful, the drug received its first regulatory approval worldwide from China's National Medical Products Administration in July for adults and adolescents aged 16 and older with narcolepsy type 1.

It is China's first approved oral medicine that selectively activates orexin receptor 2, according to Takeda, offering a treatment targeting a core disease mechanism.

Chinese researchers participated in the medicine's global development. Zhan Shuqin, a professor and a chief physician in the neurology department at Xuanwu Hospital, took part in Phase I research and served as a lead investigator in Phase III and long-term extension studies.

Narcolepsy type 1 is a chronic neurological disorder caused by a deficiency of orexin, a chemical that helps regulate sleep and wakefulness. Patients can experience a range of daytime and nighttime symptoms, including excessive daytime sleepiness, disrupted nighttime sleep, and cataplexy — a sudden loss of muscle tone. They may also experience cognitive difficulties.

Oveporexton selectively stimulates the orexin receptor 2 to compensate for deficient orexin signaling. Zhan said this mechanism provided a new treatment option, with clinical studies showing improvements across the range of narcolepsy type 1 symptoms assessed, as well as benefits in patients' daily functioning and quality of life.

Two global Phase III trials — FirstLight and RadiantLight — showed significant improvements in wakefulness, daytime sleepiness, and cataplexy compared with placebo after 12 weeks, Takeda said. The most common adverse events included insomnia, urinary urgency, and frequent urination.

Zhan said timely recognition and accurate diagnosis were essential to helping patients benefit from innovative therapies. She called for individualized treatment and regular follow-up, alongside efforts to shorten the time between the onset of symptoms and diagnosis.