Takeda announced that the US Food and Drug Administration (FDA) approved ORZEYFUL (oveporexton), an oral orexin receptor 2 (OX2R) agonist, 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 US to treat the disease holistically rather than individual symptoms.

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

NT1 is a rare, chronic neurological disease driven by a loss of orexin and affects an estimated 120,000 people in the US. 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.

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

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 symptoms of the disease. These included improvements in excessive daytime sleepiness, cataplexy and health-related quality of life.

Dr. Emmanuel Mignot, MD, PhD, principal US investigator in the ORZEYFUL phase 3 program, said, “Until now, people have managed narcolepsy type 1 with treatments that target symptom relief. 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.”

Oveporexton was generally well-tolerated with a safety profile consistent across clinical studies to date. The most common side effects include trouble sleeping (insomnia), urinary urgency, urinary frequency and excessive saliva.

Andy Plump, MD, PhD, President, Research and Development (R&D), Takeda, said, "With this approval, we are turning scientific discovery into reality for adults living with narcolepsy type 1. 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.”