Takeda launches ORZEYFUL (oveporexton) in Japan and China for narcolepsy type 1

Regulatory status and launch

ORZEYFUL (oveporexton), an oral orexin receptor 2 (OX2R) agonist discovered by Takeda scientists, was approved by China’s NMPA, the U.S. FDA and Japan’s MHLW within a month and is now available in Japan and China for treatment of narcolepsy type 1 (NT1). China’s approval covers adolescents aged 16 and older and adults; the U.S. and Japan approvals cover adults. Takeda reports a U.S. launch is scheduled next month pending DEA scheduling, and global regulatory submissions are ongoing.

Mechanism and indication

ORZEYFUL is designed to selectively stimulate OX2R to restore orexin signaling and address the underlying orexin deficiency in NT1, aiming to promote wakefulness and reduce REM-like phenomena such as cataplexy, as reflected in the approved label.

Clinical data and safety

Landmark Phase 3 studies demonstrated consistent, clinically meaningful and statistically significant improvements compared with placebo across wakefulness, sleepiness, cataplexy, disease severity and quality of life measures (p<0.001) over 12 weeks, with effects observed at the earliest assessed timepoints and sustained through study duration. ORZEYFUL was generally well tolerated; the most common adverse events reported were insomnia, urinary urgency, urinary frequency and excessive saliva.