CAMBRIDGE, Mass.–Takeda announced that ORZEYFUL (oveporexton), a first-in-class oral medication for adults with narcolepsy type 1, is now available by prescription in the United States, introducing a new treatment approach that targets the underlying orexin deficiency associated with the neurological disorder.
The drug is the first approved orexin receptor agonist for narcolepsy type 1 (NT1), a chronic condition characterized by excessive daytime sleepiness, sudden muscle weakness known as cataplexy, cognitive difficulties and disrupted nighttime sleep.
Unlike existing treatments that primarily address individual symptoms, ORZEYFUL is designed to restore orexin signaling by selectively activating orexin receptor 2 (OX2R). Orexin is a neurotransmitter involved in regulating wakefulness and sleep.
According to Takeda, clinical trials demonstrated improvements across multiple symptoms of NT1, including daytime sleepiness and cataplexy, offering a new therapeutic option for patients managing the condition.
“Clinical trials showed ORZEYFUL improved the full range of symptoms, and now we can bring those results into real-world practice,” said Emmanuel Mignot, M.D., Ph.D., principal U.S. investigator in the ORZEYFUL Phase 3 program. “Using ORZEYFUL to target the underlying cause of narcolepsy type 1 is a welcome and exciting development in sleep medicine. I can now start a new conversation with my patients about their symptoms and how using this new treatment approach may make a real difference for them.”
Narcolepsy type 1 is a rare neurological disorder associated with the loss of orexin-producing neurons in the brain. The condition affects an estimated 3 million people worldwide, including approximately 120,000 in the United States.
Patients frequently experience excessive daytime sleepiness, unpredictable sleep episodes, sudden losses of muscle tone and fragmented nighttime sleep. These symptoms can interfere with employment, education, personal relationships and everyday activities.
The condition also remains significantly underdiagnosed. Takeda estimates that only about half of people living with NT1 in the United States have received a diagnosis, with the diagnostic process taking an average of 10 years.
More than half of patients receiving treatment continue to experience symptoms, highlighting the limitations of existing therapeutic approaches.
“Living with narcolepsy type 1 is an ongoing struggle to keep it together – from scheduling naps and anticipating sleep attacks to figuring out how to be present as a mom and a professional,” said Emily P., a clinical trial participant with NT1. “With ORZEYFUL, I feel more confident planning my day because my narcolepsy has become more manageable.”
ORZEYFUL works by selectively stimulating OX2R receptors, helping restore signaling normally provided by orexin. This mechanism promotes wakefulness and reduces abnormal rapid eye movement sleep-related symptoms, including cataplexy.
The medication is intended to address both daytime and nighttime symptoms associated with NT1, although individual responses may vary.
The most commonly reported side effects include insomnia, urinary urgency, increased urinary frequency and excessive saliva.
“Bringing ORZEYFUL, a first-in-class medicine, to people living with narcolepsy type 1 in the U.S. is one of those moments,” said Rhonda Pacheco, President of Takeda’s U.S. Business Unit. “We’re incredibly proud to be here, and equally focused on what comes next: supporting care and access for patients prescribed ORZEYFUL.”
The medication is available through prescriptions issued by licensed healthcare providers and will be distributed nationwide through Accredo Specialty Pharmacy, part of Evernorth Health Services.
Takeda has also established patient support programs to help eligible adults navigate insurance coverage requirements, understand treatment access procedures and explore available financial assistance options.
In addition to introducing ORZEYFUL, Takeda is developing diagnostic tools and biomarker-based approaches intended to improve the identification of narcolepsy type 1 and distinguish it from other sleep-wake disorders.
The company said the launch represents a new treatment option for adults with NT1 by addressing the biological deficiency associated with the disease rather than focusing exclusively on symptom management.
