CAMBRIDGE, Mass. — Takeda announced the simultaneous launch of ORZEYFUL (oveporexton) in Japan and China for the treatment of narcolepsy type 1, introducing a new class of medication designed to address the underlying biological cause of the neurological disorder.
The launches follow regulatory approvals within a month of one another from China’s National Medical Products Administration, the U.S. Food and Drug Administration and Japan’s Ministry of Health, Labour and Welfare.
Takeda plans to launch ORZEYFUL in the United States next month, pending completion of Drug Enforcement Administration scheduling requirements. The company is also pursuing regulatory approvals in additional markets worldwide.
ORZEYFUL is a first-in-class oral orexin receptor 2 agonist developed to restore orexin signaling in patients with narcolepsy type 1, a rare, chronic neurological condition caused by a deficiency of the brain chemical orexin.
The disorder is characterized by excessive daytime sleepiness, cataplexy, or sudden loss of muscle tone, disrupted nighttime sleep, sleep paralysis and cognitive difficulties. These symptoms can significantly affect daily activities, employment, education and quality of life.
“We’re excited at the potential to fundamentally change how NT1 is managed across Japan and China,” said Giles Platford, president of Takeda’s international business.
“We’ve heard from patients how ORZEYFUL has improved their daily functioning and quality of life, enormously meaningful outcomes for a condition known to impact many aspects of a person’s life, including work, education and social interactions,” Platford added.
Unlike existing treatments that primarily manage symptoms, ORZEYFUL is designed to address the underlying orexin deficiency by activating orexin receptor 2, helping promote wakefulness and reduce symptoms such as cataplexy.
In Phase 3 clinical trials, ORZEYFUL demonstrated statistically significant improvements compared with placebo across multiple measures, including wakefulness, daytime sleepiness, cataplexy, disease severity and quality of life.
The improvements were observed at the earliest assessment points and maintained throughout the 12-week studies, according to Takeda.
The medication was generally well tolerated, with the most commonly reported side effects including insomnia, urinary urgency, increased urinary frequency and excessive saliva.
Discovered by Takeda scientists in Japan, ORZEYFUL is the lead product in the company’s orexin-focused drug development portfolio, which includes additional programs targeting sleep-wake disorders and other conditions involving orexin biology.
Takeda said the launches represent an important step in expanding access to treatments that target the biological mechanisms of narcolepsy type 1 rather than relying exclusively on symptom management.
