Takeda’s ORZEYFUL (oveporexton) approved in China as first OX2R agonist for narcolepsy type 1

Regulatory clearance and scope

The National Medical Products Administration approved ORZEYFUL (oveporexton) for treatment of narcolepsy type 1 (NT1) in adolescents aged 16 and older and adults. Oveporexton is described as the first oral orexin receptor 2 (OX2R)-selective agonist approved in China and, per the announcement, the only approved medicine to treat NT1 in the country. The U.S. submission is under priority review with Breakthrough Therapy designation and the Japanese submission is under Sakigake designation; additional regulatory filings are planned.

Disease context

Narcolepsy is a chronic neurological disorder driven by orexin deficiency; the company estimates about 700,000 people in China have narcolepsy, with NT1 comprising roughly 75–80% of cases. NT1 typically begins between ages 10 and 20 and produces daytime and nighttime symptoms including excessive daytime sleepiness and cataplexy, with associated risks such as traffic accidents and elevated burden of anxiety and depression.

Clinical evidence

Approval was based on global Phase 3 trials FirstLight (TAK-861-3001; n=168) and RadiantLight (TAK-861-3002; n=105), randomized, placebo-controlled studies of 12 weeks' duration that enrolled across 19 countries. Both studies reported statistically significant improvements versus placebo on prespecified endpoints including measures of excessive daytime sleepiness and cataplexy; secondary endpoints included ESS, WCR, NSS-CT, PVT, PGI‑C, FINI and SF‑36.

Safety and development notes

Oveporexton demonstrated a safety profile consistent with prior data; the most common adverse events were insomnia, urinary urgency and urinary frequency. The molecule was internally discovered at Takeda and is positioned as the lead OX2R agonist in the company’s orexin development program.