Takeda has received FDA approval for ORZEYFUL (oveporexton), the first and only medicine indicated to treat the underlying cause of narcolepsy type 1 in adults. The oral orexin receptor 2 agonist is approved for narcolepsy type 1, also known as narcolepsy with cataplexy, and is designed to restore orexin signaling that is deficient in the disorder.
Takeda said adults taking Orzeyful in clinical trials experienced significant and meaningful improvements across the full range of narcolepsy type 1 symptoms compared with placebo, including excessive daytime sleepiness, cataplexy and health-related quality of life. The approval is based on data from the global Phase 3 FirstLight and RadiantLight studies, which together formed a comprehensive clinical program. Oveporexton was generally well tolerated, with the most common side effects including insomnia, urinary urgency, urinary frequency and excessive saliva.
Julie Kim, Takeda’s president and CEO, called the approval “a new chapter” for the narcolepsy type 1 community, saying the first-in-class orexin treatment has the potential to redefine how the disease is managed. Patient advocate Julie Flygare said the decision is a historic moment that expands treatment choices and offers hope for people living with the condition.
Takeda said Orzeyful enables a “different kind of conversation” in the doctor’s office by treating the disease holistically rather than focusing on individual symptoms. The company is preparing for a U.S. launch and expects the Drug Enforcement Administration to issue a controlled substance classification within 90 days. Once scheduling is complete, Orzeyful will be made available through specialty pharmacies to U.S. healthcare providers and adults with narcolepsy type 1.
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