Takeda (TSE:4502/NYSE:TAK) announced  that ORZEYFUL (oveporexton) is now available in Japan and China for the treatment of narcolepsy type 1 (NT1), providing first access to clinicians to prescribe treatment with the new class of medicine. ORZEYFUL is a first-in-class orexin receptor 2 (OX2R) agonist, in tablet form, designed to treat the underlying orexin deficiency associated with NT1.

We’re excited at the potential to fundamentally change how NT1 is managed across Japan and China.

“We’re excited at the potential to fundamentally change how NT1 is managed across Japan and China,” said Giles Platford, president, International, Takeda. “We’ve heard from patients how ORZEYFUL has improved their daily functioning and quality of life, enormously meaningful outcomes for a condition known to impact many aspects of a person’s life, including work, education and social interactions.”^1,2,3,4,5

NT1 is a chronic, rare neurological disease driven by orexin deficiency. People experience a range of symptoms including excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), disrupted nighttime sleep, sleep paralysis, hallucinations and cognitive symptoms. Many patients experience challenges that may affect their quality of life and limit their independence. People with narcolepsy have approximately twice the rate of healthcare resource utilization compared with controls, including more frequent emergency department visits, hospitalizations, outpatient appointments and physician consultations.^6,7,8

ORZEYFUL is the first therapy designed to restore orexin signaling and address the biological cause of NT1. This mechanism represents a potential paradigm shift from symptom management to a disease-targeted treatment. Landmark Phase 3 studies in people with NT1 demonstrated ORZEYFUL improved the full range of NT1 symptoms and quality of life compared with placebo⁹. Results showed consistent, clinically meaningful and statistically significant improvements with ORZEYFUL compared to placebo in wakefulness, sleepiness, cataplexy, disease severity and quality of life measures (p-values of <0.001) across doses over 12 weeks. These improvements were observed at the earliest assessed timepoint for each measure and were sustained throughout the duration of the studies. ORZEYFUL was generally well-tolerated with a safety profile consistent across clinical studies to date. The most common side effects include insomnia, urinary urgency, urinary frequency and excessive saliva.