CAMBRIDGE, Mass. — Takeda announced that Japan’s Ministry of Health, Labour and Welfare has approved ORZEYFUL (oveporexton) for the treatment of narcolepsy type 1 in adults.
ORZEYFUL is a first-in-class oral orexin receptor 2 agonist designed to address the underlying orexin deficiency associated with narcolepsy type 1, rather than treating individual symptoms. The drug was discovered in Takeda’s laboratories in Japan, and the company said it is preparing for its launch.
“Our discovery of the first orexin agonist is a successful representation of Japan-originated science that will directly impact people around the world living with narcolepsy type 1,” said Julie Kim, President and Chief Executive Officer of Takeda. “With the potential to redefine narcolepsy type 1 care, ORZEYFUL is the first validation of our broader orexin strategy, demonstrating how scientific innovation can create new possibilities for patients and drive future growth for Takeda.”
Narcolepsy type 1 is a chronic, rare neurological disorder caused by orexin deficiency. Symptoms can include excessive daytime sleepiness, cataplexy, disrupted nighttime sleep, sleep paralysis, hallucinations and cognitive difficulties.
The condition can affect work, education and social interactions. Takeda said limited awareness and the complexity of symptoms can contribute to misdiagnosis, with patients experiencing an average diagnostic delay of more than 10 years.
“NT1 is a condition that can have a significant impact on patients’ lives around the clock. Historically, treatment has primarily focused on managing individual symptoms. In recent years, advances in our understanding of the underlying pathophysiology of narcolepsy have led to the development of new therapeutic approaches,” said Yuichi Inoue, Professor, Department of Somnology, Tokyo Medical University. “I look forward to seeing a broader range of treatment options become available, helping physicians tailor treatment to the needs of each individual patient.”
The approval was supported by Takeda’s clinical development program, including the global Phase 3 FirstLight and RadiantLight studies. The trials showed statistically significant improvements with oveporexton across the range of narcolepsy type 1 symptoms evaluated, including excessive daytime sleepiness and cataplexy.
Takeda said oveporexton was generally well tolerated, with a safety profile consistent across clinical studies. The most commonly reported adverse events included insomnia, urinary urgency, urinary frequency and excessive salivation.
“We credit our scientists in Japan who worked tirelessly to successfully discover a way to target the underlying orexin deficiency associated with narcolepsy type 1, a scientific feat that had never been achieved before,” said Asuka Miyabashira, President, Japan Pharma Business Unit at Takeda. “It’s exciting for a biopharmaceutical company to discover, develop and commercialize an asset internally, and we are proud to have worked with our colleagues, healthcare providers and patient community to bring this new class of treatment to adults living with narcolepsy type 1 in Japan.”
Japan represents the third major regulatory approval for ORZEYFUL. The medicine is also approved for the treatment of narcolepsy type 1 in China and the United States. In the U.S., its controlled substance classification remains under review by the Drug Enforcement Administration.
