FDA approves Orzeyful for adults with narcolepsy type 1

The U.S. Food and Drug Administration approved Orzeyful (oveporexton) tablets for adults with narcolepsy type 1, the agency said in a press release published Aug. 5. FDA said it is the first medicine approved for the disorder as a complete condition and the first to directly target orexin signaling.

Narcolepsy type 1 is a lifelong sleep disorder with no earlier treatment aimed at its underlying biology, according to FDA. The agency said the disease affects an estimated 1 in 2,000 people in the United States and can cause excessive daytime sleepiness, cataplexy, sleep paralysis, hallucinations at sleep-wake transitions and disrupted nighttime sleep.

FDA said narcolepsy type 1 is caused by the loss of brain cells that produce orexin, a chemical messenger that helps regulate wakefulness, sleep and muscle tone. Orzeyful is an oral tablet taken twice daily, and the approval was granted to Takeda Pharmaceuticals America, Inc.

The agency said the drug’s effectiveness and safety were evaluated in two randomized, double-blind, placebo-controlled 12-week studies that enrolled 273 adults with narcolepsy type 1. In patients taking the 2 mg dose, FDA said the drug improved the ability to stay awake, reduced daytime sleepiness and lowered the number of cataplexy episodes compared with placebo. The agency also said patients reported improvements in sleep paralysis, hallucinations and disrupted nighttime sleep.

FDA said the most common side effects were insomnia, increased urinary frequency, urgency to urinate and increased saliva production. The agency said the rate of treatment discontinuation because of side effects was low.

In a statement cited in the release, Tiffany R. Farchione, director of the Division of Psychiatry in FDA’s Center for Drug Evaluation and Research, said people with narcolepsy type 1 have long had treatments that only address pieces of the condition. She said the new drug is the first medicine that impacts the underlying biology of the disease and treats narcolepsy type 1 as a whole.

FDA said Orzeyful should not be used with strong CYP3A inhibitors, and that safety and effectiveness have not been established in patients under 18. The agency also said the drug has been recommended for scheduling under the Controlled Substances Act and would be lawful to market after a Drug Enforcement Administration scheduling decision.

The approval followed the drug’s Breakthrough Therapy Designation and Priority Review, FDA said. The agency described Breakthrough Therapy Designation as a status for medicines showing early clinical evidence of substantial improvement over available therapies for serious conditions, and Priority Review as a shorter review process for drugs that could offer significant improvements in treatment.

Sources

• FDA press release: FDA approves first drug to treat full range of narcolepsy type 1 symptoms

Editorial note: This article was prepared with AI assistance and includes source attribution for reader review.