Takeda announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved the use of ORZEYFUL (oveporexton) for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults.

ORZEYFUL is a first-in-class oral orexin receptor 2 (OX2R) agonist and the only medicine indicated in Japan to treat the disease holistically rather than individual symptoms. The discovery of this new class of medicine originated in Takeda's laboratories in Japan.

Takeda is proceeding with launch preparations and expects to make ORZEYFUL available as quickly as possible.

Julie Kim, President and Chief Executive Officer (CEO), Takeda, said, “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. 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.”

NT1 is a chronic, rare neurological disease driven by orexin deficiency. People experience a range of daytime and nighttime symptoms including excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), disrupted nighttime sleep, sleep paralysis, hallucinations and cognitive symptoms. The persistent, 24-nature of the disease can severely impact many aspects of a person’s life, including work, education and social interactions. Despite the substantial disease impact, limited awareness and the complexity of symptoms can contribute to misdiagnoses and an average diagnostic delay of over 10 years.

Yuichi Inoue, Professor, Department of Somnology, Tokyo Medical University, said, “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. 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 is based on a comprehensive clinical program including the global phase 3 FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) studies that showed oveporexton offers statistically significant improvements across the full range of NT1 symptoms assessed.

These included improvements in excessive daytime sleepiness, cataplexy and other secondary measures evaluated in the studies. Oveporexton was generally well-tolerated with a safety profile consistent across clinical studies to date. The most common adverse events were trouble sleeping (insomnia), urinary urgency, urinary frequency and excessive saliva.

Asuka Miyabashira, President, Japan Pharma Business Unit, Takeda, said, “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. It’s exciting for a biopharmaceutical company to discover, develop and commercialise 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.”

The MHLW approval marks the third approval of ORZEYFUL around the world. ORZEYFUL is also approved for the treatment of NT1 in China as well as the US where the controlled substance classification for ORZEYFUL is currently under review by the US Drug Enforcement Administration (DEA).