FDA approves first drug for full range of narcolepsy symptoms

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The U.S. Food and Drug Administration has approved Orzeyful (oveporexton) tablets to treat narcolepsy type 1 in adults. Orzeyful marks the first medication approved for the full range of symptoms associated with narcolepsy type 1 and is the first approved drug to directly target the loss of orexin signaling that causes the disease.

The approval is based on two randomized, double-blind, placebo-controlled studies enrolling 273 adults with narcolepsy type 1. Over 12 weeks, patients taking Orzeyful (2 mg) in both studies showed improvements in their ability to stay awake during the day compared with those assigned to placebo.

Patients also reported a significant reduction in cataplexy episodes, as well as meaningful improvements in sleep paralysis, hallucinations and disrupted nighttime sleep. Insomnia, increased urinary frequency, urgency to urinate and increased saliva production were the most commonly reported side effects.

"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, from the FDA Center for Drug Evaluation and Research, said in a statement. "This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole."

Approval of Orzeyful was granted to Takeda Pharmaceuticals.

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