ORZEYFUL (oveporexton) Approved in Japan as the First and Only Medicine to Treat the Underlying Cause of Narcolepsy Type 1

Discovered by Takeda in Japan, ORZEYFUL has the Potential to Redefine Care Beyond Individual Symptom Management for Adults Living with Narcolepsy Type 1 (NT1) Landmark Phase 3 Studies Demonstrated Significant and Meaningful Improvements Across the Full Range of NT1 Symptoms Evaluated in Clinical Trials Compared to Placebo Milestone Marks Third Major Regulatory Approval Secured for ORZEYFUL Around the World

Takeda (TOKYO:4502/NYSE:TAK) announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) 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.

“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”

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 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 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. Learn more about the Phase 3 data results here.

“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.”

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 United States (U.S.) where the controlled substance classification for ORZEYFUL is currently under review by the U.S. Drug Enforcement Administration (DEA).

About ORZEYFUL (Oveporexton)

ORZEYFUL (oveporexton) is an oral orexin receptor 2 (OX2R) agonist, which selectively stimulates the OX2R to restore signaling and address the underlying orexin deficiency associated with narcolepsy type 1 (NT1). By activating OX2Rs, ORZEYFUL promotes wakefulness and reduces abnormal rapid eye movement (REM)-sleep like phenomena, including cataplexy (sudden and temporary loss of muscle tone), to address a range of daytime and nighttime symptoms as evaluated in clinical studies and consistent with the approved label.

About Takeda’s Orexin Franchise

Takeda is the leader in orexin science with a tailored portfolio of investigational orexin agonists in pre-clinical and clinical stages for multiple-sleep wake disorders and other indications where orexin plays a role including respiration, mood and metabolism. Oveporexton is the lead orexin receptor 2 (OX2R) agonist in Takeda’s orexin franchise and has been approved by regulatory bodies in China for the treatment of narcolepsy type 1 (NT1) in adolescents aged 16 and older and adults as well as in the United States and Japan for adults with NT1. The company is also investigating other oral orexin agonists, including TAK-360 for the treatment of NT1, narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH), as well as TAK-495.

About Takeda

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.

ORZEYFUL (oveporexton) Product Overview in Japan

Product Name:
Orzeyful Tablet 0.5mg, 1mg, 2mg

Generic Name:
Oveporexton

Indications and Effects:
Narcolepsy Type 1

Dosage and administration:
The usual adult dosage of Oveporexton is 1mg administered orally two times daily. The first dose should be taken upon awakening and the second dose 3 to 5 hours later. The dose may be increased to 2 mg oral administration two times daily only when it is well tolerated and sufficient efficacy is not observed.

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