The US Food and Drug Administration has approved oveporexton (Orzeyful) tablets for the treatment of narcolepsy type 1 in adults. The agency said Orzeyful is the first approved treatment for narcolepsy type 1 that addresses the disorder's full spectrum of symptoms.

Narcolepsy type 1 is a rare, lifelong neuropsychiatric sleep disorder caused by the loss of brain cells that produce orexin, a chemical messenger that regulates wakefulness, sleep, and muscle tone. According to the US Food and Drug Administration (FDA), patients with narcolepsy type 1 experience excessive daytime sleepiness, cataplexy, sleep paralysis, hallucinations at the transition between sleep and wakefulness, and disrupted nighttime sleep.

Oveporexton is an oral tablet administered twice daily, activating the same brain receptor normally stimulated by orexin.

The FDA evaluated the effectiveness and safety of oveporexton in two randomized, double-blind, placebo-controlled 12-week studies involving 273 adults with narcolepsy type 1. Patients who received oveporexton 2 mg had improved ability to stay awake during the day compared with patients who received placebo. Patients receiving oveporexton also reported less daytime sleepiness, a significant reduction in cataplexy episodes, and improvements in sleep paralysis, hallucinations, and disrupted nighttime sleep.

“For too long, people with narcolepsy type 1 have had to manage a complex, lifelong neuropsychiatric condition with treatments that only address pieces of it,” Tiffany R. Farchione, MD, Director of the Division of Psychiatry within the FDA Center for Drug Evaluation and Research, said in the press release. “This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole.”

The FDA reported insomnia, increased urinary frequency, urgency to urinate, and increased saliva production as the most common side effects. Treatment discontinuation because of side effects occurred infrequently.  Because oveporexton should not be used with strong CYP3A inhibitors, the FDA advises patients to discuss all current medications with their health care professionals.

Oveporexton was designated a Breakthrough Therapy and received Priority Review. The FDA said oveporexton has been recommended for scheduling under the Controlled Substances Act and may be marketed after the Drug Enforcement Agency issues a scheduling decision. The approval was granted to Takeda Pharmaceuticals America, Inc.

Source: US Food and Drug Administration