Takeda announced that FDA has approved Orzeyful (oveporexton) for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults.

The persistent, 24-hour nature of NT1 is driven by orexin deficiency and can severely impact people's lives. As a first-in-class orexin treatment, Orzeyful is the only medicine indicated in the U.S. to treat the disease holistically rather than targeting individual symptoms.¹

"With this approval, we are turning scientific discovery into reality for adults living with narcolepsy type 1," said Andy Plump, M.D., Ph.D., president of research and development at Takeda. "Orzeyful is just the beginning. With orexin emerging as a powerful therapeutic pathway, Takeda is unlocking the potential of this new class of medicine for patients across a range of disorders."

What is Orzeyful?

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 1.¹ By activating OX2Rs, Orzeyful promotes wakefulness and reduces abnormal rapid eye movement (REM)-sleep-like phenomena, including cataplexy, to address a range of daytime and nighttime symptoms as evaluated in clinical studies and consistent with the approved label.¹

What is the approval based on?

The approval is based on a comprehensive clinical program, including the global Phase III FirstLight and RadiantLight studies.¹ The FirstLight and RadiantLight studies displayed that oveporexton offers statistically significant improvements across the full range of symptoms of the disease, including improvements in excessive daytime sleepiness, cataplexy and health-related quality of life.² In the studies, Oveporexton was generally well-tolerated, with a safety profile remaining consistent across clinical studies to date.² The most common side effects include trouble sleeping (insomnia), urinary urgency, urinary frequency and excessive saliva.²

"Until now, people have managed narcolepsy type 1 with treatments that target symptom relief," said Dr. Emmanuel Mignot, M.D., Ph.D., principal U.S. investigator in the Orzeyful Phase III program. "As the first and only approved orexin therapy to treat the broad spectrum of the disease, Orzeyful can enable a different kind of conversation in the doctor's office about treatment options."

What is narcolepsy type 1?

NT1 is a rare, chronic neurological disease driven by a loss of orexin that affects an estimated 120,000 people in the U.S.¹ People with NT1 experience excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), cognitive symptoms and disrupted nighttime sleep that can severely impact multiple aspects of daily life, including work, education and social interactions.¹

"The FDA approval of Orzeyful marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment," said Julie Kim, president and chief executive officer of Takeda. "We are proud to have discovered and developed this orexin treatment innovation and will bring it to adults living with NT1 as quickly as possible."

What's next for Orzeyful?

The controlled substance classification for Orzeyful is currently under review by the Drug Enforcement Administration (DEA) and is expected within 90 days.¹ After the controlled substance classification is determined, Orzeyful is expected to be made available through a specialty pharmacy to U.S. healthcare providers and adults living with NT1.¹

The FDA approval is not expected to have a significant impact on Takeda's full year consolidated financial forecast for the fiscal year ending March 31, 2027.

"For those of us living with narcolepsy type 1, symptoms reshape everyday life and extend far beyond what most people understand about the condition," said Julie Flygare, president and chief executive officer at Project Sleep and a person with NT1. "Orzeyful's approval is a historic moment that expands our treatment choices and gives me great hope for the future and for our community."

Sources

1. U.S. FDA Approves Takeda’s ORZEYFUL™ (oveporexton), the First and Only Medicine to Treat the Underlying Cause of Narcolepsy Type 1 Takeda August 5, 2026, https://www.takeda.com/newsroom/newsreleases/2026/orzeyful-approved-narcolepsy/
2. New Pivotal Study Data Show Takeda’s Oveporexton Improved Daily Function, Cognition and Nighttime Sleep for People with Narcolepsy Type 1 Takeda June 15, 2026 https://www.takeda.com/newsroom/newsreleases/2026/oveporexton-phase-3-narcolepsy-type-1/