FDA Approves First Drug to Treat the Full Range of Narcolepsy Type 1 Symptoms

The U.S. FDA approved Orzeyful (oveporexton) tablets for treating narcolepsy type 1 in adults. It is the first medicine approved for narcolepsy type 1 as a complete disorder and the first to directly target the loss of orexin signaling that causes the disease. Narcolepsy type 1 is a rare neuropsychiatric sleep disorder affecting approximately 1 in 2,000 people in the United States, characterized by excessive daytime sleepiness, cataplexy, sleep paralysis, and hallucinations. The drug's effectiveness and safety were evaluated in two 12-week randomized, double-blind, placebo-controlled studies involving 273 adults with narcolepsy type 1. Patients taking Orzeyful 2 mg showed improvements in daytime wakefulness and significant reductions in cataplexy episodes and other narcolepsy symptoms. The most common side effects were insomnia and increased urinary frequency. The medication is taken as an oral tablet twice daily.