China's National Medical Products Administration approved an innovative drug for narcolepsy type 1 on Tuesday, making China the first country to authorize the drug for market use.
The drug is a selective orexin receptor 2 agonist intended for patients aged 16 and above. As a first-in-class therapy with a novel target and mechanism of action, it works by restoring orexin signaling and is considered the first treatment designed to address the underlying cause of narcolepsy type 1.
Narcolepsy is a rare, chronic neurological disorder. Narcolepsy type 1, which is accompanied by cataplexy, is primarily caused by the extensive loss of orexin-producing neurons in the hypothalamus, leading to a range of neurological dysfunctions.
Developed as a treatment for a rare disease, the drug was submitted for marketing approval simultaneously in several countries and regions. In China, it was granted breakthrough therapy designation and included in the priority review and approval program.
Experts said the drug’s first approval in China reflects a shift in the research and registration strategies of multinational pharmaceutical companies, from launching products overseas before introducing them to China to conducting global development programs and filing applications in China at the same time.