Takeda has received approval in Japan for ORZEYFUL (oveporexton), a new oral treatment for adults with narcolepsy type 1 (NT1), a rare neurological disorder that causes excessive daytime sleepiness and sudden loss of muscle control.
Japan’s Ministry of Health, Labour and Welfare (MHLW) approved ORZEYFUL as a treatment for NT1, also known as narcolepsy with cataplexy. The drug is a first-in-class orexin receptor 2 (OX2R) agonist and is designed to target the underlying orexin deficiency linked to the disease.
Takeda said it is preparing for the launch of ORZEYFUL in Japan and plans to make the treatment available as soon as possible.
Narcolepsy type 1 is caused by a loss of orexin, a brain chemical that helps regulate wakefulness and sleep. People with NT1 can experience a wide range of symptoms throughout the day and night, including excessive daytime sleepiness, cataplexy, disrupted nighttime sleep, sleep paralysis, hallucinations and problems with concentration.
The condition can have a major effect on work, education and social life. However, limited awareness and the wide range of symptoms can make diagnosis difficult. Patients can face an average diagnostic delay of more than 10 years.
Unlike treatments that focus on individual symptoms, ORZEYFUL is intended to address NT1 more broadly by targeting the orexin system. Takeda said the medicine could provide a new approach to managing the disease.
The Japanese approval was supported by data from Takeda’s global Phase 3 clinical program, which included the FirstLight and RadiantLight studies. Results showed statistically significant improvements across several symptoms of NT1.
The studies reported improvements in excessive daytime sleepiness, cataplexy and other measures related to the disease. Oveporexton was generally well tolerated, with its safety profile remaining consistent across clinical studies.
The most commonly reported side effects included insomnia, urinary urgency, increased urinary frequency and excessive saliva.
Takeda said the discovery of orexin agonists came from research conducted at its laboratories in Japan. The company described the approval as an important milestone for its broader orexin research strategy and for Japan-originated drug discovery.
Julie Kim, president and chief executive officer of Takeda, said the approval represents an example of Japanese scientific research that could have an impact on people living with NT1 worldwide.
The Japanese approval is the third regulatory approval for ORZEYFUL globally. The treatment has also been approved for NT1 in China and the United States. In the US, the controlled substance classification for ORZEYFUL is currently being reviewed by the Drug Enforcement Administration.
Takeda expects the Japan approval to expand treatment options for adults living with narcolepsy type 1 and provide a new way to address the underlying biology of the disease.