Health · 5 Aug
FDA approves Orzeyful, the first drug treating narcolepsy type 1 as a whole disorder
The FDA approved Orzeyful (oveporexton) tablets for narcolepsy type 1 in adults — the first medicine approved for the condition as a complete disorder and the first to act on the orexin system. Narcolepsy type 1 is caused by the loss of the brain cells that produce orexin, the messenger that regulates wakefulness, sleep and muscle tone; instead of masking individual symptoms with stimulants or sedatives, Orzeyful directly activates the same receptor orexin normally stimulates. It is taken as an oral tablet twice a day and was developed by Takeda.
Approval rests on two randomized, double-blind, placebo-controlled 12-week trials enrolling 273 adults with narcolepsy type 1. Patients on the 2 mg dose stayed awake better during the day, reported substantially less daytime sleepiness, had significantly fewer cataplexy episodes, and improved across the full range of symptoms including sleep paralysis, hallucinations and disrupted nighttime sleep. The most common side effects were insomnia, more frequent and urgent urination, and increased saliva production, and few participants stopped treatment because of side effects.
Caveats worth noting: the evidence covers only 12 weeks in a modest sample, the drug has not been studied in anyone under 18, and it should not be taken with strong CYP3A inhibitors. It has been recommended for scheduling under the Controlled Substances Act, so it can only be marketed after the DEA scheduling decision. Anyone with narcolepsy type 1 should ask their sleep specialist whether it fits their situation.