U.S. Food and Drug Administration Accepts New Drug Application and Grants Priority Review for Takeda’s Oveporexton (TAK-861) as a Potential First-in-Class Therapy for Narcolepsy Type 1

Key Terms

new drug application regulatory

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

priority review regulatory

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

narcolepsy type 1 medical

A chronic neurological disorder that causes overwhelming daytime sleepiness and sudden loss of muscle control (cataplexy) because the brain no longer keeps the body reliably awake. Think of the brain’s wakefulness switch failing, so people can unexpectedly nod off or briefly collapse during strong emotions. Investors care because it defines a clear patient population and ongoing need for treatments, diagnostics and long-term care, shaping potential market size, regulatory approval priorities and reimbursement decisions.

orexin receptor 2 (OX2R) medical

Orexin receptor 2 (OX2R) is a protein on certain brain cells that acts like a lock for naturally occurring orexin molecules that help control sleep, wakefulness, appetite and alertness; drugs act as keys to either activate or block it. Investors watch OX2R because medicines that target it can treat conditions such as narcolepsy, insomnia and other central nervous system disorders, so clinical trial results, patent coverage and safety profiles can strongly affect a drug developer’s value.

prescription drug user fee act (pdufa) regulatory

The Prescription Drug User Fee Act (PDUFA) is a law that allows drug companies to pay fees to the government to help speed up the review process for new medicines. This funding aims to ensure that important drugs reach patients faster, which can influence a company's ability to bring products to market efficiently. For investors, PDUFA-related decisions can impact drug approval timelines and company performance.

breakthrough therapy designation regulatory

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

phase 3 medical

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

• This FDA Acceptance is a Milestone for People Living with Narcolepsy Type 1 Who Need New and Different Treatment Options
• Oveporexton is an Orexin Agonist Designed to Restore Orexin Signaling and Address the Underlying Orexin Deficiency that Causes Narcolepsy Type 1
• The Prescription Drug User Fee Act (PDUFA) Target Action Date is the Third Quarter of this Calendar Year

OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) and granted Priority Review for oveporexton (TAK-861) for the treatment of narcolepsy type 1 (NT1). Oveporexton is an investigational oral orexin receptor 2 (OX2R)-selective agonist designed to address the underlying orexin deficiency that causes NT1 by restoring orexin signaling. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date in the third quarter of this calendar year. Takeda remains on track to potentially bring the first approved orexin agonist treatment to people living with NT1.

NT1 is a chronic, rare neurological disease caused by a loss of orexin and characterized by excessive daytime sleepiness and cataplexy (sudden loss of muscle tone). This results in a spectrum of physical, cognitive and psychosocial effects that can have a debilitating impact on many aspects of a person’s life, including work, education and social interactions. Despite existing therapies, the majority of patients continue to experience symptoms and are forced to cope with the continued impact of NT1.

“The FDA’s acceptance of our NDA is a milestone for people living with narcolepsy type 1,” said Andy Plump, M.D., Ph.D., president of R&D at Takeda. “Considering the high unmet need, this community deserves a new and different treatment approach that aims to address the underlying orexin deficiency that causes NT1 by restoring orexin signaling. We are one step closer to potentially transforming the current treatment paradigm and intend to deliver through our leading work in orexin science.”

The NDA filing is supported by a comprehensive data package including the FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002) global Phase 3 studies. Key oveporexton data measuring objective and patient-reported improvements in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, overall quality of life and daily life functions demonstrate statistically significant and clinically meaningful improvements achieving near normal ranges across the broad range of symptoms investigated. Oveporexton was generally well-tolerated with a safety profile consistent across clinical studies to date. The most common adverse events were insomnia, urinary urgency and urinary frequency. Learn more about the Phase 3 data results here.

Oveporexton previously received Breakthrough Therapy designation for the treatment of excessive daytime sleepiness in NT1 from the U.S. FDA and the Center for Drug Evaluation of China’s National Medical Products Administration. Oveporexton has also received Sakigake designation from the Japanese Ministry of Health, Labour and Welfare.

The NDA filing has no significant impact on the full year consolidated forecast for the fiscal year ending March 31, 2026.

About Takeda’s Orexin Franchise

Takeda is spearheading orexin science with the most advanced development program. The tailored portfolio of investigational orexin agonists could benefit a broad range of conditions where orexin biology plays a role. Oveporexton is the lead investigational orexin receptor 2 (OX2R)-selective agonist asset in Takeda’s orexin franchise, currently in late-stage development for the treatment of NT1. TAK-360 is the next oral OX2R agonist in Takeda’s orexin franchise, initially being developed for individuals with narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH). Additional orexin agonists are also in development, including 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.

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View source version on businesswire.com: https://www.businesswire.com/news/home/20260210380286/en/

Japanese Media:

Tsuyoshi Tada

tsuyoshi.tada@takeda.com

U.S. and International Media:

Cassie Ercanbrack

cassie.ercanbrack@takeda.com

Source: Takeda Pharmaceutical Company Limited