The U.S. FDA has granted approval to Takeda for Orzeyful (oveporexton), the first medication to treat narcolepsy type 1 (NT1) by targeting the cause of the disease.
Orzeyful is an oral orexin receptor 2 (OX2R) agonist. By stimulating OX2R, it treats orexin deficiency, therefore increasing wakefulness and reducing abnormal REM phenomena. This provides a holistic therapy rather than treating individual symptoms.
The approval is supported by the phase 3 FirstLight and RadiantLight trials, where adult patients saw significant and clinically-meaningful changes compared to those taking a placebo. The medication led to improvements in symptoms including excessive daytime sleepiness, cataplexy, and health-related quality of life. The drug was well tolerated and had a safety profile consistent with other clinical studies.
Orzeyful is currently undergoing the DEA scheduling process for controlled substance classification, which is expected to take no longer than 90 days.