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Key takeaways:
- ORZEYFUL (oveporexton) has secured regulatory approvals in Japan, China, and the US as the first oral orexin receptor 2 (OX2R) agonist for narcolepsy type 1.
- The medication is designed to treat the underlying orexin deficiency that drives the disease, rather than managing individual symptoms.
- Phase 3 clinical trials demonstrated statistically significant improvements in excessive daytime sleepiness, cataplexy, and other secondary measures compared to placebo.
Takeda recently secured major regulatory milestones in Asia for ORZEYFUL (oveporexton), an oral orexin receptor 2 (OX2R) agonist indicated for the treatment of narcolepsy type 1. The drug selectively stimulates the OX2R to restore signaling and address the underlying orexin deficiency that causes the chronic neurological disease.
In Japan, where the medication was originally discovered in Takeda’s laboratories, the Japanese Ministry of Health, Labour and Welfare approved its use for adults in August.
“NT1 is a condition that can have a significant impact on patients’ lives around the clock. Historically, treatment has primarily focused on managing individual symptoms,” says Yuichi Inoue, MD, professor at the department of somnology at Tokyo Medical University, in a release. “I look forward to seeing a broader range of treatment options become available, helping physicians tailor treatment to the needs of each individual patient.”
In July, the National Medical Products Administration of China approved the therapy for adolescents aged 16 years and older and adults. Narcolepsy affects an estimated 700,000 people in China, with narcolepsy type 1 accounting for approximately 75% to 80% of cases, according to Takeda.
“For years, treatment options for narcolepsy type 1 have largely relied on symptomatic therapies to manage individual symptoms,” says Han Fang, director of the sleep medicine center at Peking University People’s Hospital and director of the Peking University Sleep Research Center, in a release. “The approval of oveporexton represents a meaningful paradigm shift in narcolepsy type 1 treatment, signaling a transition from partial symptom management to a mechanism-driven approach that can help address the many symptoms evaluated in clinical studies and may support improvements in daily functioning and quality of life.”
These Asian milestones occurred at a similar time as the US Food and Drug Administration approval of the drug for adults in August.
“Until now, people have managed narcolepsy type 1 with treatments that target symptom relief,” says Emmanuel Mignot, MD, PhD, principal US investigator in the ORZEYFUL Phase 3 program, in a release. “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.”
The approvals are based on data from the Phase 3 FirstLight and RadiantLight studies, which evaluated the efficacy, safety, and tolerability of oveporexton compared to placebo over 12 weeks. The trials demonstrated statistically significant improvements on prespecified endpoints, including excessive daytime sleepiness and cataplexy. The most common adverse reactions reported were insomnia, urinary urgency, urinary frequency, and excessive saliva.
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