FDA Approves Oveporexton for Adults With Narcolepsy Type 1
Key Takeaways for Clinical Practice
- Per FDA approval announcement, oveporexton (ORZEYFUL) is approved for adults with narcolepsy type 1 (NT1). Approval was based on two phase 3 randomized, double-blind, placebo-controlled 12-week trials.
- In both trials, oveporexton improved excessive daytime sleepiness, cataplexy, and health-related quality of life versus placebo.
- Oveporexton is an oral orexin receptor 2 agonist. Common adverse events included insomnia, urinary urgency/frequency, and excessive salivation.
The US Food and Drug Administration (FDA) has approved oveporexton (ORZEYFUL), a first-in-class orexin treatment for narcolepsy type 1 (NT1) in adults. The FDA had previously granted the drug Breakthrough Therapy designation and Priority Review.
What Is Oveporexton?
The oral orexin receptor 2 (OX2R) agonist directly targets the orexin deficiency underlying the 24-hour character of NT1 that causes symptoms like daytime sleepiness, cataplexy, cognitive symptoms, nighttime sleep interruptions, and hallucinations. An estimated 120,000 people in the US live with the disease, many of whom are undiagnosed.
“ORZEYFUL is the only medicine indicated in the US to treat the disease holistically rather than individual symptoms,” said manufacturer Takeda in a press release that announced the approval.
Phase 3 Trial Results
This approval was based on two phase 3 randomized, double blind, placebo-controlled 12-week studies: FirstLight (TAK-861-3001) and RadiantLight (TAK-861-3002). Both studies demonstrated that oveporexton outperformed placebo, providing statistically significant improvements in excessive daytime sleepiness, cataplexy, and health-related quality of life measures.
In these studies, oveporexton was well-tolerated and returned a consistent safety profile. Common side effects included insomnia, urinary urgency and frequency, and excessive salivation.
Availability
Distribution of oveporexton is anticipated to begin following the conclusion of controlled substance classification by the Drug Enforcement Administration. The drug will be made available to healthcare providers and patients with NT1 via a specialty pharmacy.
“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.”
Other oral orexin agonists under investigation at Takeda include TAK-360 and TAL-495 for the treatment of NT1, narcolepsy type 2, and idiopathic hypersomnia.
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